KEYWORD INDEX AAV Vectors AAV Vectors 724 Adenovirus Vectors 102 Age-Related Macular Degeneration 189 Aging 619 Alzheimer’s Disease 59 Amyotrophic Lateral Sclerosis 606 Animal Model 629 Animal Models 50, 52, 54, 55, 56, 84, 96, 145, 168, 180, 186, 189, 223, 227, 256, 296, 337, 346, 350, 356, 361, 362, 376, 377, 498, 503, 604, 612, 613, 615, 619, 698, 721, 725, 760 Antibody Response 77 Auditory Diseases 269 Autoimmune Disease 84 Axon Regeneration 271 Barcoding 542 Brain Delivery 618 Butyrylcholinesterase 60 Cancer - Cell Therapy 678 Cancer - Gene Therapy 215, 219, 323, 548, 669, 672 Cancer - Targeted Therapy 89, 219, 723 Cancer - T-Cell Therapy 323 Capsid Engineering 252 Capsid Modification 95 Cardiovascular Diseases 89, 180, 297 CCR5 43 CD40L, X-HIGM, Gene Editing 316 CD8+ T Cells, Ovalbumin 79 Cell Delivery 554 Cell Reprogramming 266 Cell Therapy - Hematopoietic 123, 285 Cell Therapy - Mesenchymal 524, 554, 632 Cell Therapy - Muscle 85 Cell Therapy - Neuronal 99, 678 Chimeric Antigen Receptors 251 Chromatography 100 Clinical Gene Therapy 24, 83, 185, 248, 249, 297, 387, 470, 480, 497, 547, 592, 615, 697, 715, 755 Continuous Flow Ultracentrifugation 97 CRISPR/Cas 569 CRISPR/Cas9 39, 43, 119, 120, 266, 293, 303, 321, 322, 481, 482, 543, 570 Crude Lysate 290 Cytotoxic Transgene 215 Database Development 591 Delivery Techniques 29 Diabetes 50, 543 DMD Dogs 721 DNA Viral Vectors 87, 97, 102, 253, 254, 294, 306, 311, 319, 549, 630, 727 Enteric Nervous System, Gastrointestinal Disorders 612 Epigenetic Regulation 8 Evolution and Development 389 Exon Skipping 624 Exosomes 595 Functional MRI (fMRI), Visual Pathways, Brain 265 Gene Correction/Modification/Targeting 39, 43, 44, 48, 86, 93, 119, 120, 123, 129, 133, 165, 188, 295, 303, 311, 319, 359, 481, 482, 483, 485, 542, 543, 569, 570, 605, 620, 725 Molecular Therapy Volume 24, Supplement 1, May 2016 Copyright © The American Society of Gene & Cell Therapy
Gene Expression 10, 11, 60, 91, 98, 99, 158, 159, 164, 252, 270, 292, 299, 310, 370, 456, 471, 474, 545, 546, 608, 610, 616, 618, 622, 700, 701, 726, 728 Gene Therapy 94 Genetic Diseases 27, 28, 45, 49, 57, 83, 101, 164, 165, 257, 265, 269, 300, 348, 350, 381, 481, 551, 606, 617, 621, 755 Glaucoma 188 GMP Manufacturing 460, 710 Good Manufacturing Procedures 100, 308, 309, 460, 463, 591, 592, 710 Hearing 269 Hematologic Diseases 227, 760 Hematopoietic Stem Cells 285, 316 Hemophilia 52, 78, 92, 223, 227, 253, 296, 456, 545, 550, 684, 688, 760 High Quality Grade 592 HIV 44 HSV Vectors 94 Human Immune Response to AAV 24 Humoral Immune Response 82 Hybrid Vector 672 Immunotherapy 81, 196, 472, 555, 688, 699 Induced Pluripotent Stem Cells 553, 556 Infectious Diseases 44, 79, 145, 539, 555, 699 Insulin Therapy 550 Intrathecal Delivery 724 Iron-Overload 220 Krabbe Disease 57 Lentiviral Vectors 322 Lentivirus Vectors 129, 305, 632, 728 Liver 120 Long Term Expression 24, 50, 60, 305, 390, 424 Luekocyte Adhesion Deficiency 285 Lysosomal Storage Disease 348 Lysosomal Storage Diseases 361 Metabolic Disease 356 Metabolic Diseases 48, 58, 86, 93, 157, 158, 159, 162, 168, 218, 346, 349, 351, 352, 359, 366, 372, 382, 468, 611, 696 Methylmalonic Acidemia 168 MicroRNA 220, 379, 606, 669, 737, 739 Minicircle 306 MPS1 93 Mucopolysaccharidosis 485 Muscular Dystrophy 726 Musculo-Skeletal Disorders 49, 322, 378, 379, 380, 381, 382, 387, 480, 483, 497, 503, 552, 622, 624, 629, 630, 722, 726 Natural History and Trial Development 715 Neuritin 371 Neurological Disorders 27, 30, 45, 54, 55, 56, 57, 58, 59, 157, 162, 186, 252, 255, 256, 271, 295, 301, 321, 337, 348, 349, 351, 352, 356, 361, 362, 366, 368, 370, 371, 372, 376, 540, 583, 604, 605, 610, 611, 612, 613, 616, 618, 619, 621, 724 Neuromuscular Disorders 85, 90, 377, 380, 389, 390, 498, 551, 552, 629, 715, 721 Neurotrophic Factor, Parkinson 610 Novel Capsid 256
S321
KEYWORD INDEX Ophthalmic Diseases 28, 29, 31, 80, 83, 88, 96, 98, 188, 189, 248, 249, 265, 268, 270, 306, 544, 553, 556, 607, 608, 617, 620, 739 Optogenetic Therapy 270 Optogenetics 186 Orbital Shaken Bioreactors 463 Osteoporosis, Alpha-1 Antitrypsin 632 Oversized Vector 92 Passive Immunization 699 Pharmacology/Toxicology 30, 31, 88, 268, 299, 365, 368, 472, 550 Pitx3, Mitochondria, Antioxidants, Transcription 376 Pompe Disease 49, 381 Precision Medicine 7 Pre-Existing AAV Antibodies 698 Promoterless Vector 48 Promoters 99 Prostate Cancer 669 QC/QA Assays 34, 290, 467 Re-Administration 472 Regulation 546 Retina 544 RNAi and shRNA 145, 583, 737 Routes of Administration 300 SERCaMP, ATV6, ER Stress 218 Single-Molecule, Real-Time Sequencing 7 Somatic-Transgenic Technology 305 Spinal Muscular Atrophy, Type 1 480 Sporadic Inclusion Body Myositis, Follistatin 497 TALENs 316, 569 Targeted Gene Expression 29, 298, 300, 307, 478, 542, 695, 723 Targeting 251 Thermostability 34 Tissue Engineering 524, 702 TLR9 Signaling 696 Toxicology Studies 365, 468, 470 Transgene Engineering 456 Urea Cycle Disorder 45 Vaccines 196, 722 Vector Characterization/Biology 7, 8, 9, 10, 11, 12, 34, 82, 91, 218, 254, 255, 257, 293, 298, 301, 302, 304, 307, 389, 474, 478, 540, 541, 546, 547, 548, 607, 695, 727 Vector Immunology/Host Responses 77, 78, 79, 80, 81, 82, 90, 185, 310, 378, 424, 467, 595, 696, 697, 698, 700, 701, 702 Vector Production 9, 12, 87, 92, 94, 95, 97, 100, 101, 290, 292, 294, 302, 304, 308, 309, 460, 463, 471, 539, 549, 591, 672, 684, 710 Vector Purification 294 Viral Evolution 541 Wet Age-Related Macular Degeneration 248 XLRP 101 Zinc-Finger Nucleases 133, 485 AAV Animal Models Gene Expression Genetic Diseases Metabolic Diseases Musculo-Skeletal Disorders Neurological Disorders S322
499 499 25 25 499 25
Adenovirus Cytokine Cancer - Gene Therapy 405 Cancer - T-Cell Therapy 405 Immunotherapy 405 Adenovirus Vectors AAV Vectors 102 Animal Models 16, 417, 519 Apoptosis 211, 659 Breast Cancer 417 Cancer - Cell Therapy 117, 420 Cancer - Gene Therapy 16, 113, 117, 413, 414, 417, 418, 421, 461, 509, 514, 516, 598, 659, 662 Cancer - Targeted Therapy 13, 103, 105, 211, 408, 421, 517, 519, 639, 655, 661 Cancer - T-Cell Therapy 410, 513 Cardiovascular Diseases 179 Cell Delivery 420 Cell Therapy - Hematopoietic 757 Cell Therapy - Mesenchymal 453 Checkpoint Inhibitor 513 Clinical Gene Therapy 36, 109, 110, 112, 115, 116, 453, 514, 661 CRISPR/Cas9 15, 104 DNA Viral Vectors 13, 14, 17, 102, 106 Gene Correction/Modification/Targeting 15, 18 Gene Expression 109, 110, 112, 113, 115, 116, 324, 413 Genetic Diseases 47 Good Manufacturing Procedures 706, 713 Hematopoietic Stem Cells 106, 757 Immunotherapy 408, 410, 414, 418, 509, 513, 516, 639, 642, 662 Infectious Diseases 18, 22, 103 Keloid 112 Long Term Expression 324, 598 Mesothelioma 211, 661 Metabolic Diseases 47, 179, 484 Musculo-Skeletal Disorders 104 Novel Molecules to Enhance Viral Entry 176 Pancreatic Cancer 639 Pharmacology/Toxicology 176 QC/QA Assays 461 Respiratory/Lung Disorder 22 Respiratory/Lung Disorders 176 RNAi and shRNA 105 Tumor Microenvironment 662 Vaccines 22, 36, 642 Vector Characterization/Biology 14, 17, 655 Vector Immunology/Host Responses 517 Vector Production 706, 713 Zinc-Finger Nucleases 484 Adrenoleukodystrophy Clinical Gene Therapy Lentivirus Vectors Neurological Disorders
250 250 250
Age-Related Macular Degeneration AAV Vectors Animal Models Ophthalmic Diseases
189 189 189
Molecular Therapy Volume 24, Supplement 1, May 2016 Copyright © The American Society of Gene & Cell Therapy
KEYWORD INDEX Aging AAV Vectors Animal Models Neurological Disorders
619 619 619
Alzheimer’s Disease AAV Vectors 59 CRISPR/Cas9 567 Gene Correction/Modification/Targeting 567 Neurological Disorders 59, 567 Amyotrophic Lateral Sclerosis AAV Vectors Genetic Diseases MicroRNA
606 606 606
Animal Model AAV Vectors Musculo-Skeletal Disorders Neuromuscular Disorders
629 629 629
Animal Models AAV 499 AAV Vectors 50, 52, 54, 55, 56, 84, 96, 145, 168, 180, 186, 189, 223, 227, 256, 296, 337, 346, 350, 356, 361, 362, 376, 377, 498, 503, 604, 612, 613, 615, 619, 698, 721, 725, 760 Adenovirus Vectors 16, 417, 519 Age-Related Macular Degeneration 189 Aging 619 Animal Models 84 Apoptosis 411 Autoimmune Disease 84 Bone Defect 631 Breast Cancer 417 Cancer - Gene Therapy 16, 205, 412, 417, 667, 673 Cancer - Targeted Therapy 411, 412, 415, 519, 660, 674 Cancer - T-Cell Therapy 275 Cardiopulmonary 383 Cardiovascular Diseases 180, 447, 448 Cell Therapy - Hematopoietic 427 Cell Therapy - Lung 182 Cell Therapy - Mesenchymal 448 Cell Therapy - Muscle 635 Cell Therapy - Other 430, 447 Chimeric Antigen Receptors 750 Clinical Gene Therapy 383, 615 Colorectal Cancer 675 CRISPR/Cas9 167 Cytokines 441 Diabetes 50 DMD Dogs 721 Enteric Nervous System, Gastrointestinal Disorders 612 Esophageal Squamous Cell Carcinoma 412 Ethical Issues 627 Gastrointestinal Inflammation 477 Gene Correction/Modification/Targeting 38, 284, 725 Gene Expression 64, 441, 477, 499, 675, 749 Genetic Diseases 166, 183, 350, 357, 383, 631 Hematologic Diseases 227, 760 Hematopoietic Stem Cells 758, 767 Hemophilia 52, 223, 227, 296, 492, 756, 760 Molecular Therapy Volume 24, Supplement 1, May 2016 Copyright © The American Society of Gene & Cell Therapy
HIV 427 HSV Vectors 656, 660, 667 Immunotherapy 63, 64, 275, 427, 693, 740, 749, 750 Infectious Diseases 145, 173, 431, 437, 767 Injectable Biologic 26 Insertional Mutagenesis 2 Lentivirus Vectors 2, 431, 631, 674, 756 Liver Diseases 183 Long Term Expression 50, 693 Lysosomal Storage Deseases, Enzyme Replacement 430 Lysosomal Storage Diseases 361 Metabolic Disease 356 Metabolic Diseases 166, 168, 346, 357, 430, 477 Methylmalonic Acidemia 168 MicroRNA 529, 738 Musculo-Skeletal Disorders 388, 499, 503, 633, 635 Neurological Disorders 26, 54, 55, 56, 186, 256, 337, 356, 361, 362, 376, 604, 612, 613, 619, 627, 738 Neuromuscular Disorders 377, 388, 498, 623, 633, 721 Non-Invasive 633 Non-Viral Gene Delivery 173, 490, 492 Notch Signaling 447 Novel Capsid 256 Oligonucleotide Based Therapies 580, 623, 740 Oncolytic Virus 411 Ophthalmic Diseases 96, 189 Optogenetics 186 Pitx3, Mitochondria, Antioxidants, Transcription 376 Pneumonia, Sepsis, Lung Contusion 173 Pre-Existing AAV Antibodies 698 Reovirus 415 Respiratory/Lung Disorders 167, 182, 183, 490, 580 Retrovirus Vectors 284 RNA Viral Vectors 205, 529, 673 RNAi and shRNA 145 TALENs 758 Targeted Gene Expression 26, 675 T-Cell Populations 693 Therapeutic Gene Modulation 656 Ultrasound 492 Vector Characterization/Biology 2 Vector Immunology/Host Responses 63, 415, 437, 698 Zinc-Finger Nucleases 38 Anti CD19 CAR T KTE-C19 Cancer - Cell Therapy Cancer - T-Cell Therapy Chimeric Antigen Receptors
745 745 745
Antibody Delivery Hematopoietic Stem Cells Infectious Diseases Long Term Expression
429 429 429
Antibody Response AAV Vectors Vector Immunology/Host Responses Antisense Oligonucleotides Infectious Diseases Oligonucleotide Based Therapies
77 77 586 586
S323
KEYWORD INDEX Anti-Transgene Ophthalmic Diseases Vector Immunology/Host Responses
694 694
Apoptosis Adenovirus Vectors 211, 659 Animal Models 411 Cancer - Gene Therapy 149, 345, 659 Cancer - Targeted Therapy 211, 411 Chemical Conjugates 345 Curcumin, CDC, Breast Cancer 345 Mesothelioma 211 Oncolytic Virus 411 RNA Delivery 149 Sendai Virus 149 Aptamers Bioinformatics 148 Cancer - Targeted Therapy 671 Oligonucleotide Based Therapies 148, 744 Pharmacology/Toxicology 671 RNA Delivery 148, 744 Assay Development Clinical Gene Therapy 171 Gene Expression 171 Hemophilia 171 Auditory Diseases AAV Vectors 269 Genetic Diseases 269 Hearing 269 Autoimmune disease AAV Vectors Animal Models
84 84
Axon Regeneration AAV Vectors Neurological Disorders
271 271
Barcoding AAV Vectors Gene Correction/Modification/Targeting Targeted Gene Expression
542 542 542
Bioinformatics Aptamers 148 Clinical Gene Therapy 537 Oligonucleotide Based Therapies 148 Precision Medicine 537 RNA Delivery 148 RNA Viral Vectors 537 Bioprocess Cell Processing Cell Reprogramming Non-Viral Gene Delivery
343 343 343
Bone Defect Animal Models Genetic Diseases Lentivirus Vectors
631 631 631
S324
Brain Delivery AAV Vectors Gene Expression Neurological Disorders
618 618 618
Breast Cancer Adenovirus Vectors Animal Models Cancer - Gene Therapy
417 417 417
Butyrylcholinesterase AAV Vectors Gene Expression Long Term Expression
60 60 60
Cancer - Cell Therapy AAV Vectors 678 Adenovirus Vectors 117, 420 Anti CD19 CAR T KTE-C19 745 Cancer - Gene Therapy 66, 117, 192, 207 Cancer - Targeted Therapy 66, 204, 209, 216, 393 Cancer - T-Cell Therapy 192, 199, 204, 393, 402, 515, 646, 652, 745, 748 Cancer Stem Cells, Tumor Microenvironment 679 Cell Delivery 242, 420, 442, 511 Cell Processing 402, 459 Cell Reprogramming 645 Cell Therapy - ES Cells and iPS cells 528 Cell Therapy - Hematopoietic 233, 273 Cell Therapy - Mesenchymal 242 Cell Therapy - Muscle 636 Cell Therapy - Neuronal 678 Cell Therapy - Other 645 Chimeric Antigen Receptor T Cells 204 Chimeric Antigen Receptors 207, 515, 745, 748 CRISPR/Cas9 422 Gene Correction/Modification/Targeting 641 Gene Expression 216, 679 Good Manufacturing Procedures 459 Hematologic Diseases 233, 276 Hematopoietic Stem Cells 273 Immunotherapy 199, 276, 422, 469, 476, 511, 646, 652 Integrase Defective Lentivirus Quantification 469 Lentivirus Vectors 212, 462, 469, 476 Musculo-Skeletal Disorders 636 Nanobiotechnology 209, 442 Neurological Disorders 528 Oligonucleotide Based Therapies 263 Oncolytic Adenovirus 515 Pharmacology/Toxicology 476 QC/QA Assays 263 Reactive Oxygen Species 216 Respiratory/Lung Disorders 212 RNA Viral Vectors 462 Stem Cells; Ovarian Cancer; Drug Delivery 209 Targeted Gene Expression 679 Telomerase 276 Zinc-Finger Nucleases 641 Cancer - Gene Therapy AAV Vectors Adenovirus Cytokine
215, 219, 323, 548, 669, 672 405 Molecular Therapy Volume 24, Supplement 1, May 2016 Copyright © The American Society of Gene & Cell Therapy
KEYWORD INDEX Adenovirus Vectors
16, 113, 117, 413, 414, 417, 418, 421, 461, 509, 514, 516, 598, 659, 662 Animal Models 16, 205, 412, 417, 667, 673 Apoptosis 149, 345, 659 Breast Cancer 417 Cancer - Cell Therapy 66, 117, 192, 207 Cancer - Targeted Therapy 66, 155, 217, 219, 412, 416, 419, 421, 658, 670, 677, 762 Cancer - T-Cell Therapy 74, 192, 206, 323, 405, 506, 653, 746, 747, 752, 762 Cell Therapy - Hematopoietic 523 Cell Therapy - Lung 585 Chemical Conjugates 345, 602 Chimeric Antigen Receptor, CAR 670 Chimeric Antigen Receptors 74, 207, 747 Clinical Gene Therapy 61, 514 Colorectal Cancer 664 Curcumin, CDC, Breast Cancer 345 Cytotoxic Transgene 215 DNA Viral Vectors 419, 520, 664 Esophageal Squamous Cell Carcinoma 412 Ex-Vivo Expansion 523 Gene Expression 113, 142, 413 Hematologic Diseases 677 Hematopoietic Stem Cells 523, 668 HSV Vectors 184, 520, 658, 664, 667, 680 Hybrid Vector 672 Immunotherapy 61, 142, 201, 206, 261, 405, 414, 418, 495, 509, 516, 643, 644, 653, 662, 746 In Vivo T Cell Engineering 506 Lentivirus Vectors 214, 217, 475, 703 Long Term Expression 598 Measles Virus, Glioblastoma 62 Mesothelioma 217 MicroRNA 62, 144, 154, 587, 669 Multiple Myeloma 752 Neurological Disorders 184 Non-Small Cell Lung Cancer 585 Non-Viral Gene Delivery 152, 155, 493, 495, 593, 599, 644, 680 Non-Viral Vectorology 599 Oligonucleotide Based Therapies 261 Oncolytic Vectors 520 PDGF, Angiogenesis 152 Platelets 668 Polymers 493 Prostate Cancer 669 QC/QA Assays 461 Retroviral Replicating Vector 61 Retrovirus Vectors 403, 602 RNA Delivery 149, 593 RNA Viral Vectors 62, 201, 205, 409, 416, 506, 673, 703 RNAi and shRNA 144, 152, 154, 585, 587, 588, 599 Sendai Virus 149 Synthetic Gene Delivery Systems 493, 588 Targeted Gene Expression 214, 668 Toxicology Studies 475 Tumor Microenvironment 662 Tumor-Specific Promoter 677 Vaccines 643 Vector Characterization/Biology 409, 548 Vector Immunology/Host Responses 403 Molecular Therapy Volume 24, Supplement 1, May 2016 Copyright © The American Society of Gene & Cell Therapy
Vector Production Zinc-Finger Nucleases
672, 703 752
Cancer - Targeted Therapy AAV Vectors 89, 219, 723 Adenovirus Vectors 13, 103, 105, 211, 408, 421, 517, 519, 639, 655, 661 Animal Models 411, 412, 415, 519, 660, 674 Apoptosis 211, 411 Aptamers 671 Cancer - Cell Therapy 66, 204, 209, 216, 393 Cancer - Gene Therapy 66, 155, 217, 219, 412, 416, 419, 421, 658, 670, 677, 762 Cancer - Targeted Therapy 670 Cancer - T-Cell Therapy 202, 204, 392, 393, 396, 505, 762 Cardiovascular Diseases 89 Cell Therapy - Neuronal 208, 443 Chimeric Antigen Receptor T Cells 204 Chimeric Antigen Receptor, CAR 670 Chimeric Antigen Receptors 392 Clinical Gene Therapy 72, 661 DNA Vaccines, Electroporation 401 DNA Viral Vectors 13, 419 Esophageal Squamous Cell Carcinoma 412 Gene Expression 216 Glioma 665 Hematologic Diseases 65, 663, 677 HSV Vectors 658, 660, 665, 666 Immunotherapy 202, 401, 408, 423, 505, 639, 647, 663, 666 Infectious Diseases 103 Lentivirus Vectors 70, 217, 647, 674 Mesothelioma 211, 217, 661 MicroRNA 67 Mitochondrial Biogenesis 65 Nanobiotechnology 209, 396, 494 Neural Stem Cells, Pediatric Brain Tumors 443 Neurological Disorders 665 Non-Viral Gene Delivery 72, 155, 494 Oligonucleotide Based Therapies 262, 590 Oncolytic Virus 411 Oncolytic Viruses 67, 423, 654, 666 Pancreatic Cancer 639 Pharmacology/Toxicology 671 Polymeric, PBAE 494 Reactive Oxygen Species 216 Reovirus 415 RNA Delivery 262 RNA Viral Vectors 67, 416, 423, 654 RNAi and shRNA 70, 105 Stem Cells; Ovarian Cancer; Drug Delivery 209 Synthetic Gene Delivery Systems 590 Tandem Bispecific Chimeric Antigen Receptors 392 Targeted Gene Expression 723 Tumor-Specific Promoter 677 U1 Adaptors, iRGD, KRAS, MYC, Pancreatic Cancer 262 Vector Characterization/Biology 655 Vector Immunology/Host Responses 208, 415, 517 Cancer - T-Cell Therapy AAV Vectors Adenovirus Cytokine Adenovirus Vectors
323 405 410, 513 S325
KEYWORD INDEX Animal Models 275 Anti CD19 CAR T KTE-C19 745 Cancer - Cell Therapy 192, 199, 204, 393, 402, 515, 646, 652, 745, 748 Cancer - Gene Therapy 74, 192, 206, 323, 405, 506, 653, 746, 747, 752, 762 Cancer - Targeted Therapy 202, 204, 392, 393, 396, 505, 762 CAR T Cells; Lymphoma; Manufacturing 287 CD19, Sleeping Beauty 278 Cell Delivery 596 Cell Processing 287, 402, 455, 650, 714 Cell Therapy - ES Cells and iPS Cells 236 Cell Therapy - Hematopoietic 236 Cell Therapy - Mesenchymal 466 Checkpoint Inhibitor 513 Chimeric Antigen Receptor T Cells 204 Chimeric Antigen Receptors 69, 73, 74, 190, 193, 195, 198, 200, 203, 210, 247, 277, 392, 394, 397, 398, 400, 406, 451, 507, 510, 512, 515, 640, 649, 650, 651, 676, 745, 747, 748, 751 Clinical Gene Therapy 247, 272 Clonality, T-Cell Repertoire 232 CRISPR/Cas9 404, 572 DNA Viral Vectors 194 Gene Correction/Modification/Targeting 121, 391, 394, 398, 572 Genetic Diseases 232 Glycobiology 510 Good Manufacturing Procedures 75, 203, 466, 714 Hematologic Diseases 71, 213, 400, 637 IL13Ra2, GBM, Solid Tumors 76 IL-4 751 Immunotherapy 71, 73, 75, 76, 190, 191, 193, 194, 195, 197, 198, 199, 202, 203, 206, 232, 272, 275, 277, 278, 287, 391, 405, 406, 410, 451, 455, 505, 507, 510, 512, 513, 637, 638, 640, 646, 648, 649, 651, 652, 653, 746, 751 In Vivo T Cell Engineering 506 Inflammatory Toxicity 197 Lentiviral Vectors 195 Lentivirus Vectors 121, 404, 638, 648 Leukemia and Lymphoma 69, 210 Meganuclease, AAV vectors 394 Meganucleases 200 Multiple Myeloma 752 Mutated Neoantigens 391 Nanobiotechnology 396 Non-Viral Gene Delivery 278 Non-Viral Vectorology 213 Oncolytic Adenovirus 515 Oncolytic Virus 68 PD-1 572 Pediatric Cancers - Solid Tumors 649 Process Modeling; Monte Carlo Simulations 650 RNA Viral Vectors 68, 506 Sleeping Beauty Transposition; Minicircle 73 TALENs 397, 676 Tandem Bispecific Chimeric Antigen Receptors 392 Targeted Gene Expression 76 Vaccines 68, 191, 596 WT1 193 Zinc-Finger Nucleases 752 S326
Cancer Stem Cells, Tumor Microenvironment Cancer - Cell Therapy Gene Expression Targeted Gene Expression
679 679 679
Capsid Engineering AAV Vectors Gene Expression Neurological Disorders
252 252 252
Capsid Modification AAV Vectors Vector Production
95 95
CAR T Cells; Lymphoma; Manufacturing Cancer - T-Cell Therapy 287 Cell Processing 287 Immunotherapy 287 Cardiopulmonary Animal Models Clinical Gene Therapy Genetic Diseases
383 383 383
Cardiovascular Diseases AAV Vectors 89, 180, 297 Adenovirus Vectors 179 Animal Models 180, 447, 448 Cancer - Targeted Therapy 89 Cell Delivery 174, 178 Cell Therapy - Endothelial 174 Cell Therapy - ES Cells and iPS Cells 244 Cell Therapy - Mesenchymal 246, 448 Cell Therapy - Other 174, 178, 447 Chemical Conjugates 177 Clinical Gene Therapy 297 CRISPR/Cas9 20 Familial Hypercholesterolaemia 19 Gene Expression 172, 177 Genetic Diseases 20, 358 Long Term Expression 172 Metabolic Diseases 179, 358 MicroRNA 19 MitoTimer 358 Non-Viral Gene Delivery 19 Notch Signaling 447 QC/QA Assays 244 Tissue Engineering 246 CCR5 AAV Vectors 43 CRISPR/Cas9 43 Gene Correction/Modification/Targeting 43 CD19, Sleeping Beauty Cancer - T-Cell Therapy 278 Immunotherapy 278 Non-Viral Gene Delivery 278 CD40L, X-HIGM, Gene Editing AAV Vectors 316 Hematopoietic Stem Cells 316 TALENs 316 Molecular Therapy Volume 24, Supplement 1, May 2016 Copyright © The American Society of Gene & Cell Therapy
KEYWORD INDEX CD8+ T Cells, Ovalbumin AAV Vectors Infectious Diseases Vector Immunology/Host Responses
79 79 79
Cell Delivery AAV Vectors 554 Adenovirus Vectors 420 Cancer - Cell Therapy 242, 420, 442, 511 Cancer - T-Cell Therapy 596 Cardiovascular Diseases 174, 178 Cell Therapy - Endothelial 174 Cell Therapy - Hematopoietic 336, 521, 603 Cell Therapy - Lung 21 Cell Therapy - Mesenchymal 242, 554 Cell Therapy - Neuronal 521 Cell Therapy - Other 174, 178, 340 CRISPR/Cas9 496, 603 Duchenne Muscular Dystrophy; Exon Skipping 584 Exosomes 496 Genetic Diseases 21, 336 Immunotherapy 511 Nanobiotechnology 442 Neuromuscular Disorders 584 Oligonucleotide Based Therapies 584 Tissue Engineering 340 Tunneling Nanotubes 336 Vaccines 596 Cell Processing Bioprocess 343 Cancer - Cell Therapy 402, 459 Cancer - T-Cell Therapy 287, 402, 455, 650, 714 CAR T Cells; Lymphoma; Manufacturing 287 Cell Reprogramming 343 Cell Therapy - Hematopoietic 526 Cell Therapy - Mesenchymal 245 Cell Therapy - Neuronal 375 Chimeric Antigen Receptors 650 Genetic Diseases 425 Good Manufacturing Procedures 459, 714 GPCR, TDP-43, Cannabinoids 375 Hematopoietic Stem Cells 526 Immunotherapy 287, 425, 455 Lentivirus Vectors 707 MicroRNA 245 Non-Viral Gene Delivery 343 Pharmacology/Toxicology 375 Process Modeling; Monte Carlo Simulations 650 T-Cell Development, Infections, Notch, Thymus 425 Vector Production 707 Cell Reprogramming AAV Vectors 266 Bioprocess 343 Cancer - Cell Therapy 645 Cell Processing 343 Cell Therapy - Muscle 525 Cell Therapy - Other 645 CRISPR/Cas9 266 Gen Expression 704 Gene Correction/Modification/Targeting 40 Molecular Therapy Volume 24, Supplement 1, May 2016 Copyright © The American Society of Gene & Cell Therapy
Lentivirus Vectors Musculo-Skeletal Disorders Non-Viral Gene Delivery Targeted Gene Expression Vector Characterization/Biology Vector Production Cell Therapy - Endothelial Cardiovascular Diseases Cell Delivery Cell Therapy - Other
704 525 343, 344 40 344 704 174 174 174
Cell Therapy - ES Cells and iPS Cells Cancer - Cell Therapy 528 Cancer - T-Cell Therapy 236 Cardiovascular Diseases 244 Cell Therapy - Hematopoietic 234, 236 Cell Therapy - Muscle 565, 628 Clinical Gene Therapy 527 CRISPR/Cas9 527, 730 Gene Correction/Modification/Targeting 122, 243, 312, 332, 347, 730 Genetic Diseases 522 Hematologic Diseases 444, 522 Induced Pluripotent Stem Cells 241, 243, 444 Lentivirus Vector 234 Lentivirus Vectors 241 Lentivirus, DMD 565 Long Term Expression 234 Metabolic Diseases 347 Musculo-Skeletal Disorders 312 Neurological Disorders 528 Neuromuscular Disorders 730 Non-Viral Gene Delivery 628 QC/QA Assays 244 Respiratory/Lung Disorders 122 TALENs 332, 565 Transposon 628 Urea Cycle Disorder 347 Cell Therapy - Hematopoietic AAV Vectors 123, 285 Adenovirus Vectors 757 Animal Models 427 Cancer - Cell Therapy 233, 273 Cancer - Gene Therapy 523 Cancer - T-Cell Therapy 236 Cell Delivery 336, 521, 603 Cell Processing 526 Cell Therapy - ES Cells and iPS Cells 234, 236 Cell Therapy - Neuronal 521 Cell Therapy - Other 763 Clinical Gene Therapy 235 CRISPR/Cas9 603 Ex-Vivo Expansion 523 Foamy Virus 683 Gene Correction/Modification/Targeting 123, 432, 562 Gene Expression 766 Genetic Diseases 163, 336 Genetic Modification 734 Hematologic Diseases 233, 683, 763 S327
KEYWORD INDEX Hematopoietic Stem Cells 235, 273, 283, 285, 432, 523, 526, 562, 689, 734, 757 HIV 427, 763 Immunotherapy 239, 427, 685 Infectious Diseases 685 Lentivirus Vector 234 Lentivirus Vectors 283, 766 Long Term Expression 234, 689 Luekocyte Adhesion Deficiency 285 Macrophages, HSC 766 Metabolic Diseases 163 Neurological Disorders 374 RNA Viral Vectors 6, 374, 683 TALEN 562 Tissue Engineering 239 Tunneling Nanotubes 336 Vector Production 6 Zinc-Finger Nucleases 734 Cell Therapy - Lung Animal Models Cancer - Gene Therapy Cell Delivery Cell Therapy - Other Genetic Diseases Non-Small Cell Lung Cancer Pulmonary Macrophage Transplantation Respiratory/Lung Disorders RNA Viral Vectors RNAi and shRNA Vector Immunology/Host Responses
182 585 21 35 21 585 35 35, 182 438 585 438
Cell Therapy - Mesenchymal AAV Vectors 524, 554, 632 Adenovirus Vectors 453 Animal Models 448 Cancer - Cell Therapy 242 Cancer - T-Cell Therapy 466 Cardiovascular Diseases 246, 448 Cell Delivery 242, 554 Cell Processing 245 Cell Therapy - Muscle 238 Clinical Gene Therapy 453 Diabetes 354 Gene Correction/Modification/Targeting 354 Good Manufacturing Procedures 466 Lentivirus Vectors 632 Metabolic Diseases 354 MicroRNA 245 Musculo-Skeletal Disorders 238 Osteoporosis, Alpha-1 Antitrypsin 632 Tissue Engineering 246, 524 Cell Therapy - Muscle AAV Vectors 85 Animal Models 635 Cancer - Cell Therapy 636 Cell Reprogramming 525 Cell Therapy - ES Cells and iPS Cells 565, 628 Cell Therapy - Mesenchymal 238 Cell Therapy - Other 446 CRISPR/Cas9 577 S328
Gene Expression 449 Lentivirus Vectors 240 Lentivirus, DMD 565 Musculo-Skeletal Disorders 238, 446, 449, 525, 577, 635, 636 Neuromuscular Disorders 85 Non-Viral Gene Delivery 628 TALENs 565 Tissue Engineering 240 Transposon 628 Cell Therapy - Neuronal AAV Vectors 99, 678 Cancer - Cell Therapy 678 Cancer - Targeted Therapy 208, 443 Cell Delivery 521 Cell Processing 375 Cell Therapy - Hematopoietic 521 Gene Expression 99 GPCR, TDP-43, Cannabinoids 375 Neural Stem Cells, Pediatric Brain Tumors 443 Pharmacology/Toxicology 375 Promoters 99 Vector Immunology/Host Responses 208 Cell Therapy - Other Animal Models 430, 447 Cancer - Cell Therapy 645 Cardiovascular Diseases 174, 178, 447 Cell Delivery 174, 178, 340 Cell reprogramming 645 Cell Therapy - Endothelial 174 Cell Therapy - Hematopoietic 763 Cell Therapy - Lung 35 Cell Therapy - Muscle 446 Commercialisation 452 Commercialization 718 CRISPR/Cas9 573 Gene Correction/Modification/Targeting 573 Gene Expression 454 Genetic Diseases 161 Good Manufacturing Procedures 445 Hematologic Diseases 763 HIV 763 Immunotherapy 426 Infectious Diseases 426 Long Term Expression 161 Lysosomal Storage Deseases, Enzyme Replacement 430 Magnetic Targeting, MRI 338 Metabolic Diseases 430 MicroRNA 338 Musculo-Skeletal Disorders 446, 454 Non-Viral Gene Delivery 338 Notch Signaling 447 Ophthalmic Diseases 445 Pulmonary Macrophage Transplantation 35 Regulatory Issues 718 Respiratory/Lung Disorders 35 Tissue Engineering 340, 452 Cellular Clearance Gene Expression Ophthalmic Diseases
187 187 Molecular Therapy Volume 24, Supplement 1, May 2016 Copyright © The American Society of Gene & Cell Therapy
KEYWORD INDEX CGD Stem Cell Gene Correction CRISPR/Cas9 41 Gene Correction/Modification/Targeting 41 Non-Viral Gene Delivery 41 Checkpoint Inhibitor Adenovirus Vectors 513 Cancer - T-Cell Therapy 513 Immunotherapy 513 Chemical Conjugates Apoptosis 345 Cancer - Gene Therapy 345, 602 Cardiovascular Diseases 177 Curcumin, CDC, Breast Cancer 345 Gene Expression 177 Immunotherapy 225 Lentivirus Vectors 225 Nanobiotechnology 342 Ophthalmic Diseases 342 Retrovirus Vectors 602 T-Cells 225 Chimeric Antigen Receptor T Cells Cancer - Cell Therapy Cancer - Targeted Therapy Cancer - T-Cell Therapy
204 204 204
Chimeric Antigen Receptor, CAR Cancer - Gene Therapy Cancer - Targeted Therapy
670 670
Chimeric Antigen Receptors AAV Vectors 251 Animal Models 750 Anti CD19 CAR T KTE-C19 745 Cancer - Cell Therapy 207, 515, 745, 748 Cancer - Gene Therapy 74, 207, 747 Cancer - Targeted Therapy 392 Cancer - T-Cell Therapy 69, 73, 74, 190, 193, 195, 198, 200, 203, 210, 247, 277, 392, 394, 397, 398, 400, 406, 451, 507, 510, 512, 515, 640, 649, 650, 651, 676, 745, 747, 748, 751 Cell Processing 650 Clinical Gene Therapy 247 CRISPR/Cas9 274 Gene Correction/Modification/Targeting 394, 398, 761 Glycobiology 510 Good Manufacturing Procedures 203 Hematologic Diseases 400, 407 Hemophilia 434 IL-4 751 Immunotherapy 73, 190, 193, 195, 198, 203, 274, 277, 399, 406, 434, 451, 507, 510, 512, 640, 649, 651, 750, 751, 761 Lentiviral Vectors 195 Lentivirus Vectors 407 Leukemia and Lymphoma 69, 210 Meganuclease, AAV Vectors 394 Meganucleases 200 Non-Viral Gene Delivery 705 Oncolytic Adenovirus 515 Pediatric Cancers - Solid Tumors 649 Molecular Therapy Volume 24, Supplement 1, May 2016 Copyright © The American Society of Gene & Cell Therapy
Process Modeling; Monte Carlo Simulations 650 Regulatory T Cell 434 Sleeping Beauty Transposition; Minicircle 73 TALENs 397, 676 Tandem Bispecific Chimeric Antigen Receptors 392 Targeting 251 Toxicology Studies 399 Transposon 705 WT1 193 Chromatography AAV Vectors Good Manufacturing Procedures Vector Production
100 100 100
Chronic Pain HSV Vectors Neurological Disorders Targeted Gene Expression
111 111 111
Clinical Gene Therapy AAV Vectors 24, 83, 185, 248, 249, 297, 387, 470, 480, 497, 547, 592, 615, 697, 715, 755 Adenovirus Vectors 36, 109, 110, 112, 115, 116, 453, 514, 661 Adrenoleukodystrophy 250 Animal Models 383, 615 Assay Development 171 Bioinformatics 537 Cancer - Gene Therapy 61, 514 Cancer - Targeted Therapy 72, 661 Cancer - T-Cell Therapy 247, 272 Cardiopulmonary 383 Cardiovascular Diseases 297 Cell Therapy - ES Cells and iPS Cells 527 Cell Therapy - Hematopoietic 235 Cell Therapy - Mesenchymal 453 Chimeric Antigen Receptors 247 CRISPR/Cas9 326, 527, 731 Cystic Fibrosis 711 Gene Correction/Modification/Targeting 280, 326, 731 Gene Expression 109, 110, 112, 115, 116, 171 Genetic Diseases 83, 224, 353, 383, 534, 755 Good Manufacturing Procedures 592 HCV, AAV 258 Hematologic Diseases 279, 280 Hematopoietic Stem Cells 235, 314, 364, 754 Hemophilia 171 High Quality Grade 592 Human Immune Response to AAV 24 Immunotherapy 61, 272 Infectious Diseases 258 Integration Sites 531 Keloid 112 Lentivirus Vectors 250, 279, 364, 531, 534, 711, 754 Long Term Expression 24 Mathematical Modeling 314 Mesothelioma 661 Metabolic Diseases 353 MicroRNA 282 Musculo-Skeletal Disorders 328, 387, 480, 497 Natural History and Trial Development 715 Neurological Disorders 250, 267, 367 S329
KEYWORD INDEX Neuromuscular Disorders 715 Non-Viral Gene Delivery 72 Oligonucleotide Based Therapies 328 Ophthalmic Diseases 83, 248, 249, 267 Precision Medicine 537 Regulatory Issues 719 Retroviral Replicating Vector 61 Retrovirus Vectors 224, 367, 531 RNA Viral Vectors 537 RNAi and shRNA 258 Spinal Muscular Atrophy, Type 1 480 Sporadic Inclusion Body Myositis, Follistatin 497 Targeted Gene Expression 282 Toxicology Studies 470 Vaccines 36 Vector Characterization/Biology 547 Vector Immunology/Host Responses 185, 697 Vector Production 711 Wet Age-Related Macular Degeneration 248 Clonality, T-Cell Repertoire Cancer - T-Cell Therapy 232 Genetic Diseases 232 Immunotherapy 232 Colorectal Cancer Animal Models Cancer - Gene Therapy DNA Viral Vectors Gene Expression HSV Vectors Targeted Gene Expression
675 664 664 675 664 675
Commercialisation Cell Therapy - Other Tissue Engineering
452 452
Commercialization Cell Therapy - Other Regulatory Issues
718 718
Common Rule Ethical Issues Regulatory Issues
717 717
Continuous Flow Ultracentrifugation AAV Vectors DNA Viral Vectors Vector Production
97 97 97
CRISPR/Cas AAV Vectors 569 Gene Correction/Modification/Targeting 569 TALENs 569 CRISPR/Cas9 AAV Vectors 39, 43, 119, 120, 266, 293, 303, 321, 322, 481, 482, 543, 570 Adenovirus Vectors 15, 104 Alzheimer’s Disease 567 Animal Models 167 Cancer - Cell Therapy 422 Cancer - T-Cell Therapy 404, 572 Cardiovascular Diseases 20 S330
CCR5 43 Cell Delivery 496, 603 Cell Reprogramming 266 Cell Therapy - ES Cells and iPS Cells 527, 730 Cell Therapy - Hematopoietic 603 Cell Therapy - Muscle 577 Cell Therapy - Other 573 CGD Stem Cell Gene Correction 41 Chimeric Antigen Receptors 274 Clinical Gene Therapy 326, 527, 731 Diabetes 543 Dystrophin 561 Epigenetic Regulation 131, 385, 729 Exosomes 313, 496 Gene Correction/Modification/Targeting 3, 15, 39, 41, 43, 119, 120, 124, 126, 128, 131, 134, 136, 137, 139, 140, 303, 313, 317, 326, 329, 330, 334, 481, 482, 487, 488, 501, 504, 543, 559, 561, 567, 568, 570, 571, 572, 573, 575, 576, 729, 730, 731, 732, 733 Gene Expression 334 Genetic Diseases 20, 481, 488, 561, 568, 576, 733 Genomic Deletions 135 Hematologic Diseases 140, 559, 571, 692 Hematopoietic Stem Cells 135, 330, 564, 732, 768 Hemophilia 128 HIV Latency 125 HIV-1, Virual Escape 331 Homology-Directed Repair 568 HSC Engraftment, Hematologic Disease 732 Immunotherapy 274, 422, 768 Induced Pluripotent Stem Cells 318, 557, 626, 735 Infectious Diseases 125, 331, 574, 768 Lentiviral Vectors 322 Lentivirus Vectors 3, 126, 134, 135, 327, 331, 404, 532, 564, 574 Liver 120 Musculo-Skeletal Disorders 104, 139, 317, 322, 385, 501, 504, 577, 735 Neurological Disorders 321, 327, 567 Neuromuscular Disorders 730 Non-Viral Gene Delivery 41, 335, 487 Ophthalmic Diseases 124, 329 PD-1 572 Precision Medicine 141, 575 QC/QA Assays 141 Respiratory/Lung Disorders 23, 167 RNA Delivery 335 RNAi and shRNA 23, 532 Synthetic Biology 136 TALENs 136, 137, 139, 692 Targeted Epigenetic Editing 729 Targeted Gene Expression 125, 557, 626 Thalassemia 692 Tissue Engineering 318 Vector Characterization/Biology 293 Crude Lysate AAV Vectors QC/QA Assays Vector Production
290 290 290
Molecular Therapy Volume 24, Supplement 1, May 2016 Copyright © The American Society of Gene & Cell Therapy
KEYWORD INDEX Curcumin, CDC, Breast Cancer Apoptosis 345 Cancer - Gene Therapy 345 Chemical Conjugates 345 Cystic Fibrosis Clinical Gene Therapy Lentivirus Vectors Vector Production
711 711 711
Cytokines Animal Models Gene Expression
441 441
Cytotoxic Transgene AAV Vectors Cancer - Gene Therapy
215 215
Database Development AAV Vectors Good Manufacturing Procedures Vector Production
591 591 591
Delivery Techniques AAV Vectors Ophthalmic Diseases Targeted Gene Expression Design of Experiment Lentivirus Vectors Vector Production
29 29 29 708 708
Diabetes AAV Vectors 50, 543 Animal Models 50 Cell Therapy - Mesenchymal 354 CRISPR/Cas9 543 Gene Correction/Modification/Targeting 354, 543 Long Term Expression 50 Metabolic Diseases 354 DMD Dogs AAV Vectors Animal Models Neuromuscular Disorders
721 721 721
DNA Vaccines, Electroporation Cancer - Targeted Therapy 401 Immunotherapy 401 DNA Vaccines Non-Viral Gene Delivery 436 Vaccines 436 DNA Viral Vectors AAV Vectors 87, 97, 102, 253, 254, 294, 306, 311, 319, 549, 630, 727 Adenovirus Vectors 13, 14, 17, 102, 106 Cancer - Gene Therapy 419, 520, 664 Cancer - Targeted Therapy 13, 419 Cancer - T-Cell Therapy 194 Colorectal Cancer 664 Continuous Flow Ultracentrifugation 97 Gene Correction/Modification/Targeting 311, 319 Molecular Therapy Volume 24, Supplement 1, May 2016 Copyright © The American Society of Gene & Cell Therapy
Hematopoietic Stem Cells 106 Hemophilia 253 HSV Vectors 107, 520, 664 Immunotherapy 194 Minicircle 306 Musculo-Skeletal Disorders 630 Oncolytic Vectors 520 Ophthalmic Diseases 306 Targeted Gene Expression 108 Vaccines 712 Vector Characterization/Biology 14, 17, 107, 108, 254, 727 Vector Production 87, 97, 294, 549, 712 Vector Purification 294 Down Syndrome Genetic Diseases Neurological Disorders RNAi and shRNA
53 53 53
Duchenne Muscular Dystrophy; Exon Skipping Cell Delivery Neuromuscular Disorders Oligonucleotide Based Therapies
584 584 584
Dystrophin CRISPR/Cas9 561 Gene Correction/Modification/Targeting 561 Genetic Diseases 561 Enteric Nervous System, Gastrointestinal Disorders AAV Vectors Animal Models Neurological Disorders Epigenetic Regulation AAV Vectors CRISPR/Cas9 Gene Correction/Modification/Targeting Musculo-Skeletal Disorders Targeted Epigenetic Editing Vector Characterization/Biology Esophageal Squamous Cell Carcinoma Animal Models Cancer - Gene Therapy Cancer - Targeted Therapy
612 612 612
8 131, 385, 729 131, 729 385 729 8 412 412 412
Ethical Issues Animal Models 627 Common Rule 717 Ethical Issues 717 Gene Correction/Modification/Targeting 720 Nanobiotechnology 720 Neurological Disorders 627 Regulatory Issues 717 Evolution and Development AAV Vectors Neuromuscular Disorders Vector Characterization/Biology
389 389 389
Exon Skipping AAV Vectors Musculo-Skeletal Disorders
624 624 S331
KEYWORD INDEX Exosomes AAV Vectors Cell Delivery CRISPR/Cas9 Gene Correction/Modification/Targeting Non-Viral Gene Delivery Ophthalmic Diseases RNA Delivery Vector Characterization/Biology Vector Immunology/Host Responses Vector Transduction
595 496 313, 496 313 341 594 341 594 595 594
Ex-Vivo Expansion Cancer - Gene Therapy Cell Therapy - Hematopoietic Hematopoietic Stem Cells
523 523 523
Familial Hypercholesterolaemia Cardiovascular Diseases MicroRNA Non-Viral Gene Delivery
19 19 19
Fanconi Anemia Gene Correction/Modification/Targeting Genetic Diseases Hematopoietic Stem Cells Foamy Virus Cell Therapy - Hematopoietic Hematologic Diseases Hematopoietic Stem Cells RNA Viral Vectors Vector Characterization/Biology
753 753 753 683 683 1 1, 683 1
FSHD Musculo-Skeletal Disorders Oligonucleotide Based Therapies Targeted Gene Expression
153 153 153
Functional MRI (fMRI), Visual Pathways, Brain AAV Vectors Genetic Diseases Ophthalmic Diseases
265 265 265
Gastrointestinal Inflammation Animal Models Gene Expression Metabolic Diseases
477 477 477
Gen Expression Cell Reprogramming Lentivirus Vectors Vector Production
704 704 704
Gene Correction/Modification/Targeting AAV Vectors 39, 43, 44, 48, 86, 93, 119, 120, 123, 129, 133, 165, 188, 295, 303, 311, 319, 359, 481, 482, 483, 485, 542, 543, 569, 570, 605, 620, 725 Adenovirus Vectors 15, 18 Alzheimer’s Disease 567 Animal Models 38, 284, 725 Barcoding 542 Cancer - Cell Therapy 641 Cancer - T-Cell Therapy 121, 391, 394, 398, 572 S332
CCR5 43 Cell Reprogramming 40 Cell Therapy - ES Cells and iPS Cells 122, 243, 312, 332, 347, 730 Cell Therapy - Hematopoietic 123, 432, 562 Cell Therapy - Mesenchymal 354 Cell Therapy - Other 573 CGD Stem Cell Gene Correction 41 Chimeric Antigen Receptors 394, 398, 761 Clinical Gene Therapy 280, 326, 731 CRISPR/Cas 569 CRISPR/Cas9 3, 15, 39, 41, 43, 119, 120, 124, 126, 128, 131, 134, 136, 137, 139, 140, 303, 313, 317, 326, 329, 330, 334, 481, 482, 487, 488, 501, 504, 543, 559, 561, 567, 568, 570, 571, 572, 573, 575, 576, 729, 730, 731, 732, 733 Diabetes 354, 543 DNA Viral Vectors 311, 319 Dystrophin 561 Epigenetic Regulation 131, 729 Ethical Issues 720 Exosomes 313 Fanconi Anemia 753 Gene Expression 334, 560 Genetic Diseases 165, 226, 325, 481, 488, 558, 561, 568, 576, 579, 601, 733, 753 Glaucoma 188 Hematologic Diseases 42, 118, 140, 280, 559, 560, 571, 682 Hematopoietic Stem Cells 37, 42, 330, 432, 558, 562, 732, 753 Hemophilia 128 HIV 44 Homology-Directed Repair 568 HSC Engraftment, Hematologic Disease 732 Immunodeficiency 325 Immunotherapy 132, 391, 761 Induced Pluripotent Stem Cells 243 Infectious Diseases 18, 44, 765 Inflammation 226 Lentivirus Vectors 3, 121, 126, 127, 129, 130, 134, 226, 286, 533, 538, 566, 682, 764 Liver 120 Meganuclease, AAV Vectors 394 Meganucleases 138, 579 Metabolic Diseases 48, 86, 93, 347, 354, 359 MicroRNA 742 MPS1 93 Mucopolysaccharidosis 485 Musculo-Skeletal Disorders 139, 312, 317, 483, 501, 504 Mutated Neoantigens 391 Nanobiotechnology 563, 720 Neurological Disorders 295, 567, 605, 742 Neuromuscular Disorders 138, 730 Non-Viral Gene Delivery 41, 487, 601 Non-Viral Vectorology 533 Ophthalmic Diseases 124, 188, 329, 620 PD-1 572 Precision Medicine 575 Promoterless Vector 48 Respiratory/Lung Disorders 122 Retrovirus Vectors 284 RNA Delivery 538 Molecular Therapy Volume 24, Supplement 1, May 2016 Copyright © The American Society of Gene & Cell Therapy
KEYWORD INDEX Synthetic Biology 136 TALEN 562 TALENs 132, 136, 137, 139, 325, 332, 569 Targeted Epigenetic Editing 729 Targeted Gene Expression 40, 315, 542 Urea Cycle Disorder 347 Vector Immunology/Host Responses 764 Vector Production 286 XIST-Mediated Chromosome Silencing 315 Zinc-Finger Nucleases 37, 38, 118, 127, 130, 133, 315, 485, 563, 566, 641, 765 Gene Expression AAV 499 AAV Vectors 10, 11, 60, 91, 98, 99, 158, 159, 164, 252, 270, 292, 299, 310, 370, 456, 471, 474, 545, 546, 608, 610, 616, 618, 622, 700, 701, 726, 728 Adenovirus Vectors 109, 110, 112, 113, 115, 116, 324, 413 Animal Models 64, 441, 477, 499, 675, 749 Assay Development 171 brain Delivery 618 Butyrylcholinesterase 60 Cancer - Cell Therapy 216, 679 Cancer - Gene Therapy 113, 142, 413 Cancer - Targeted Therapy 216 Cancer Stem Cells, Tumor Microenvironment 679 Capsid Engineering 252 Cardiovascular Diseases 172, 177 Cell Therapy - Hematopoietic 766 Cell Therapy - Muscle 449 Cell Therapy - Neuronal 99 Cell Therapy - Other 454 Cellular Clearance 187 Chemical Conjugates 177 Clinical Gene Therapy 109, 110, 112, 115, 116, 171 Colorectal Cancer 675 CRISPR/Cas9 334 Cytokines 441 Gastrointestinal Inflammation 477 Gene Correction/Modification/Targeting 334, 560 Genetic Diseases 164, 373 Hematologic Diseases 560 Hemophilia 5, 171, 456, 545 Immunotherapy 64, 142, 749 Infectious Diseases 681 Inflammation 146 Insertional Mutagenesis 681 Keloid 112 Lentivirus Vectors 5, 609, 728, 766 Long Term Expression 60, 172, 324, 609 Macrophages, HSC 766 Metabolic Diseases 158, 159, 477 MicroRNA 146 Minicircle 743 Muscular Dystrophy 726 Musculo-Skeletal Disorders 449, 454, 499, 622, 726 Neurological Disorders 252, 370, 373, 610, 616, 618, 736 Neurotrophic Factor, Parkinson 610 Non-Viral Vectorology 289, 743 Oligonucleotide Based Therapies 146 Ophthalmic Diseases 98, 187, 270, 320, 608, 736 Optogenetic Therapy 270 Molecular Therapy Volume 24, Supplement 1, May 2016 Copyright © The American Society of Gene & Cell Therapy
Pharmacology/Toxicology 299 Promoters 99 Reactive Oxygen Species 216 Regulation 546 Respiratory/Lung Disorders 743 RNA Viral Vectors 681 Targeted Gene Expression 320, 675, 679 Transgene Engineering 456 Vector Characterization/Biology 10, 11, 91, 289, 474, 546 Vector Immunology/Host Responses 310, 700, 701 Vector Production 292, 471 Gene Therapy AAV Vectors HSV Vectors Vector Production
94 94 94
Genetic Diseases AAV 25 AAV Vectors 27, 28, 45, 49, 57, 83, 101, 164, 165, 257, 265, 269, 300, 348, 350, 381, 481, 551, 606, 617, 621, 755 Adenovirus Vectors 47 Amyotrophic Lateral Sclerosis 606 Animal Models 166, 183, 350, 357, 383, 631 Auditory Diseases 269 Bone Defect 631 Cancer - T-Cell Therapy 232 Cardiopulmonary 383 Cardiovascular Diseases 20, 358 Cell Delivery 21, 336 Cell Processing 425 Cell Therapy - ES Cells and iPS Cells 522 Cell Therapy - Hematopoietic 163, 336 Cell Therapy - Lung 21 Cell Therapy - Other 161 Clinical Gene Therapy 83, 224, 353, 383, 534, 755 Clonality, T-Cell Repertoire 232 CRISPR/Cas9 20, 481, 488, 561, 568, 576, 733 Down Syndrome 53 Dystrophin 561 Fanconi Anemia 753 Functional MRI (fMRI), Visual Pathways, Brain 265 Gene Correction/Modification/Targeting 165, 226, 325, 481, 488, 558, 561, 568, 576, 579, 601, 733, 753 Gene Expression 164, 373 GM2 Gangliosidosis 363 Hearing 269 Hematologic Diseases 522, 581 Hematopoietic Stem Cells 32, 291, 558, 753 Homology-Directed Repair 568 Immunodeficiency 325 Immunotherapy 232, 425 Inflammation 226 Krabbe Disease 57 Lentivirus Vectors 226, 291, 534, 631 Liver Diseases 183 Long Term Expression 161, 439 Lysosomal Storage Disease 348 Meganucleases 579 Metabolic Diseases 25, 46, 47, 160, 163, 166, 169, 353, 357, 358, 360, 369 MicroRNA 606 S333
KEYWORD INDEX MitoTimer 358 Musculo-Skeletal Disorders 49, 381, 741 Neurological Disorders 25, 27, 45, 53, 57, 160, 348, 360, 363, 369, 373, 621, 741 Neuromuscular Disorders 150, 551 Non-Viral Gene Delivery 169, 439, 597, 601 Oligonucleotide Based Therapies 150, 581, 741 Ophthalmic Diseases 28, 46, 83, 265, 617 Pompe Disease 49, 381 Respiratory/Lung Disorders 183, 597 Retrovirus Vectors 224 RNAi and shRNA 53 Routes of Administration 300 Splice, X-Linked Agammaglobulinemia 581 TALENs 325 Targeted Gene Expression 300 T-Cell Development, Infections, Notch, Thymus 425 Toxicology Studies 32 Tunneling Nanotubes 336 Urea Cycle Disorder 45 Vector Characterization/Biology 257 Vector Production 101 XLRP 101 Genetic Modification Cell Therapy - Hematopoietic Hematopoietic Stem Cells Zinc-Finger Nucleases
734 734 734
Genomic Deletions CRISPR/Cas9 135 Hematopoietic Stem Cells 135 Lentivirus Vectors 135 Glaucoma AAV Vectors Gene Correction/Modification/Targeting Ophthalmic Diseases
188 188 188
Glioma Cancer - Targeted Therapy HSV Vectors Neurological Disorders
665 665 665
Glycobiology Cancer - T-Cell Therapy 510 Chimeric Antigen Receptors 510 Immunotherapy 510 GM2 Gangliosidosis Genetic Diseases Neurological Disorders GMP Manufacturing AAV Vectors Good Manufacturing Procedures Vector Production
363 363 460, 710 460, 710 460, 710
Good Manufacturing Procedures AAV Vectors 100, 308, 309, 460, 463, 591, 592, 710 Adenovirus Vectors 706, 713 Cancer - Cell Therapy 459 Cancer - T-Cell Therapy 75, 203, 466, 714 Cell Processing 459, 714 S334
Cell Therapy - Mesenchymal 466 Cell Therapy - Other 445 Chimeric Antigen Receptors 203 Chromatography 100 Clinical Gene Therapy 592 Database Development 591 GMP Manufacturing 460, 710 High Quality Grade 592 Immunotherapy 75, 203 Ophthalmic Diseases 445 Orbital Shaken Bioreactors 463 Vector Production 100, 308, 309, 460, 463, 591, 706, 710, 713 GPCR, TDP-43, Cannabinoids Cell Processing 375 Cell Therapy - Neuronal 375 Pharmacology/Toxicology 375 HBV, AAV Infectious Diseases Long Term Expression RNAi and shRNA
260 260 260
HCV, AAV Clinical Gene Therapy Infectious Diseases MicroRNA Regulatory Issues RNAi and shRNA
258 258 143 143 143, 258
Hearing AAV Vectors Auditory Diseases Genetic Diseases
269 269 269
Hematologic Diseases AAV Vectors Animal Models Cancer - Cell Therapy Cancer - Gene Therapy Cancer - Targeted Therapy Cancer - T-Cell Therapy Cell Therapy - ES Cells and iPS Cells Cell Therapy - Hematopoietic Cell Therapy - Other Chimeric Antigen Receptors Clinical Gene Therapy CRISPR/Cas9 Foamy Virus Gene Correction/Modification/Targeting
227, 760 227, 760 233, 276 677 65, 663, 677 71, 213, 400, 637 444, 522 233, 683, 763 763 400, 407 279, 280 140, 559, 571, 692 683 42, 118, 140, 280, 559, 560, 571, 682 Gene Expression 560 Genetic Diseases 522, 581 Hematopoietic Stem Cells 4, 42, 221, 228, 229, 231, 281, 508, 759 Hemophilia 227, 760 HIV 763 Immunotherapy 71, 276, 508, 637, 663 Induced Pluripotent Stem Cells 444 Lentivirus Vectors 4, 221, 228, 229, 230, 279, 281, 407, 682, 686, 690, 759 Mitochondrial Biogenesis 65 Mixed Chimerism 231 Molecular Therapy Volume 24, Supplement 1, May 2016 Copyright © The American Society of Gene & Cell Therapy
KEYWORD INDEX Non-Viral Vectorology 213 Oligonucleotide Based Therapies 581 Pharmacology/Toxicology 690 QC/QA Assays 473 RNA Viral Vectors 683 Splice, X-Linked Agammaglobulinemia 581 TALENs 686, 692 Telomerase 276 Thalassemia 692 Tumor-Specific Promoter 677 Vector Characterization/Biology 230, 473 Zinc-Finger Nucleases 118 Hematopoietic Stem Cells AAV Vectors 285, 316 Adenovirus Vectors 106, 757 Animal Models 758, 767 Antibody Delivery 429 Cancer - Cell Therapy 273 Cancer - Gene Therapy 523, 668 CD40L, X-HIGM, Gene Editing 316 Cell Processing 526 Cell Therapy - Hematopoietic 235, 273, 283, 285, 432, 523, 526, 562, 689, 734, 757 Clinical Gene Therapy 235, 314, 364, 754 CRISPR/Cas9 135, 330, 564, 732, 768 DNA Viral Vectors 106 Ex-Vivo Expansion 523 Fanconi Anemia 753 Foamy Virus 1 Gene Correction/Modification/Targeting 37, 42, 330, 432, 558, 562, 732, 753 Genetic Diseases 32, 291, 558, 753 Genetic Modification 734 Genomic Deletions 135 Hematologic Diseases 4, 42, 221, 228, 229, 231, 281, 508, 759 HIV Stem Cell Gene Therapy 333 HSC Engraftment, Hematologic Disease 732 Immunotherapy 508, 768 Infectious Diseases 333, 429, 687, 767, 768 Lentivirus Vectors 4, 135, 221, 222, 228, 229, 281, 283, 291, 333, 364, 458, 530, 536, 564, 754, 759 Long Term Expression 429, 689 Luekocyte Adhesion Deficiency 285 Mathematical Modeling 314 Mixed Chimerism 231 Nanobiotechnology 536 Platelets 668 Retrovirus Vectors 530, 687 RNA Viral Vectors 1 TALEN 562 TALENs 316, 758 Targeted Gene Expression 668 Toxicology Studies 32 Vector Characterization/Biology 1 Vector Production 458 Viral Transduction Enhancers 536 Zinc-Finger Nucleases 37, 734 β-Thalassemias, Hemoglobinopathies 222
Molecular Therapy Volume 24, Supplement 1, May 2016 Copyright © The American Society of Gene & Cell Therapy
Hemophilia AAV Vectors
52, 78, 92, 223, 227, 253, 296, 456, 545, 550, 684, 688, 760 Animal Models 52, 223, 227, 296, 492, 756, 760 Assay Development 171 Chimeric Antigen Receptors 434 Clinical Gene Therapy 171 CRISPR/Cas9 128 DNA Viral Vectors 253 Gene Correction/Modification/Targeting 128 Gene Expression 5, 171, 456, 545 Hematologic Diseases 227, 760 Immunotherapy 434, 688 Insulin Therapy 550 Lentivirus Vectors 5, 756 Non-Viral Gene Delivery 492 Oversized Vector 92 Pharmacology/Toxicology 550 Regulatory T Cell 434 Transgene Engineering 456 Ultrasound 492 Vector Immunology/Host Responses 78 Vector Production 92, 684
High Quality Grade AAV Vectors Clinical Gene Therapy Good Manufacturing Procedures
592 592 592
HIV Latency CRISPR/Cas9 125 Infectious Diseases 125 Targeted Gene Expression 125 HIV Stem Cell Gene Therapy Hematopoietic Stem Cells Infectious Diseases Lentivirus Vectors HIV
333 333 333
AAV Vectors 44 Animal Models 427 Cell Therapy - Hematopoietic 427, 763 Cell Therapy - Other 763 Gene Correction/Modification/Targeting 44 Hematologic Diseases 763 Immunotherapy 427 Infectious Diseases 44
HIV-1, Virual Escape CRISPR/Cas9 331 Infectious Diseases 331 Lentivirus Vectors 331 Homology-Directed Repair CRISPR/Cas9 568 Gene Correction/Modification/Targeting 568 Genetic Diseases 568 HSC Engraftment, Hematologic Disease CRISPR/Cas9 732 Gene Correction/Modification/Targeting 732 Hematopoietic Stem Cells 732 S335
KEYWORD INDEX HSV Vectors AAV Vectors 94 Animal Models 656, 660, 667 Cancer - Gene Therapy 184, 520, 658, 664, 667, 680 Cancer - Targeted Therapy 658, 660, 665, 666 Chronic Pain 111 Colorectal Cancer 664 DNA Viral Vectors 107, 520, 664 Gene Therapy 94 Glioma 665 Immunotherapy 518, 666 Neurological Disorders 111, 114, 184, 657, 665 Non-Viral Gene Delivery 680 Oncolytic Vectors 520 Oncolytic Viruses 666 Retrovirus Vectors 657 Targeted Gene Expression 111, 114 Therapeutic Gene Modulation 656 Vector Characterization/Biology 107 Vector Immunology/Host Responses 518 Vector Production 94 Human Immune Response to AAV AAV Vectors Clinical Gene Therapy Long Term Expression
24 24 24
Humoral Immune Response AAV Vectors Vector Characterization/Biology Vector Immunology/Host Responses
82 82 82
Hybrid Vector AAV Vectors Cancer - Gene Therapy Vector Production
672 672 672
IL13Ra2, GBM, solid tumors Cancer - T-Cell Therapy 76 Immunotherapy 76 Targeted Gene Expression 76 IL-4
Cancer - T-Cell Therapy 751 Chimeric Antigen Receptors 751 Immunotherapy 751
Immunodeficiency Gene Correction/Modification/Targeting 325 Genetic Diseases 325 TALENs 325 Immunotherapy AAV Vectors 81, 196, 472, 555, 688, 699 Adenovirus Cytokine 405 Adenovirus Vectors 408, 410, 414, 418, 509, 513, 516, 639, 642, 662 Animal Models 63, 64, 275, 427, 693, 740, 749, 750 Cancer - Cell Therapy 199, 276, 422, 469, 476, 511, 646, 652 Cancer - Gene Therapy 61, 142, 201, 206, 261, 405, 414, 418, 495, 509, 516, 643, 644, 653, 662, 746 Cancer - Targeted Therapy 202, 401, 408, 423, 505, 639, 647, 663, 666 S336
Cancer - T-Cell Therapy 71, 73, 75, 76, 190, 191, 193, 194, 195, 197, 198, 199, 202, 203, 206, 232, 272, 275, 277, 278, 287, 391, 405, 406, 410, 451, 455, 505, 507, 510, 512, 513, 637, 638, 640, 646, 648, 649, 651, 652, 653, 746, 751 CAR T Cells; Lymphoma; Manufacturing 287 CD19, Sleeping Beauty 278 Cell Delivery 511 Cell Processing 287, 425, 455 Cell Therapy - Hematopoietic 239, 427, 685 Cell Therapy - Other 426 Checkpoint Inhibitor 513 Chemical Conjugates 225 Chimeric Antigen Receptors 73, 190, 193, 195, 198, 203, 274, 277, 399, 406, 434, 451, 507, 510, 512, 640, 649, 651, 750, 751, 761 Clinical Gene Therapy 61, 272 Clonality, T-Cell Repertoire 232 CRISPR/Cas9 274, 422, 768 DNA Vaccines, Electroporation 401 DNA Viral Vectors 194 Gene Correction/Modification/Targeting 132, 391, 761 Gene Expression 64, 142, 749 Genetic Diseases 232, 425 Glycobiology 510 Good Manufacturing Procedures 75, 203 Hematologic Diseases 71, 276, 508, 637, 663 Hematopoietic Stem Cells 508, 768 Hemophilia 434, 688 HIV 427 HSV Vectors 518, 666 IL13Ra2, GBM, Solid Tumors 76 IL-4 751 Infectious Diseases 264, 426, 428, 433, 555, 685, 699, 768 Inflammatory Toxicity 197 Integrase Defective Lentivirus Quantification 469 Lentiviral Vectors 195 Lentivirus Vectors 51, 225, 469, 476, 638, 647, 648 Long Term Expression 693 Mutated Neoantigens 391 Non-Viral Gene Delivery 264, 278, 433, 495, 644 Oligonucleotide Based Therapies 261, 740 Oncolytic Viruses 423, 666 Pancreatic Cancer 639 Passive Immunization 699 Pediatric Cancers - Solid Tumors 649 Pharmacology/Toxicology 472, 476 Re-Administration 472 Regulatory T Cell 434 Retroviral Replicating Vector 61 RNA Viral Vectors 201, 423 Sleeping Beauty Transposition ; Minicircle 73 Synthetic Gene Delivery Systems 428 TALENs 132 Targeted Gene Expression 76 T-Cell Development, Infections, Notch, Thymus 425 T-Cell Populations 693 T-Cells 225 Telomerase 276 Tissue Engineering 239 Toxicology Studies 399 Tumor Microenvironment 662 Molecular Therapy Volume 24, Supplement 1, May 2016 Copyright © The American Society of Gene & Cell Therapy
KEYWORD INDEX Vaccines 191, 196, 395, 642, 643 Vector Immunology/Host Responses 51, 63, 81, 395, 518 WT1 193 In Vivo T Cell Engineering Cancer - Gene Therapy Cancer - T-Cell Therapy RNA Viral Vectors Induced Pluripotent Stem Cells AAV Vectors Cell Therapy - ES Cells and iPS Cells CRISPR/Cas9 Gene Correction/Modification/Targeting Hematologic Diseases Lentivirus Vectors Musculo-Skeletal Disorders Ophthalmic Diseases Targeted Gene Expression Tissue Engineering
506 506 506 553, 556 241, 243, 444 318, 557, 626, 735 243 444 241 735 553, 556 557, 626 318
Infectious Diseases AAV Vectors 44, 79, 145, 539, 555, 699 Adenovirus Vectors 18, 22, 103 Animal Models 145, 173, 431, 437, 767 Antibody Delivery 429 Antisense Oligonucleotides 586 Cancer - Targeted Therapy 103 CD8+ T Cells, Ovalbumin 79 Cell Therapy - Hematopoietic 685 Cell Therapy - Other 426 Clinical Gene Therapy 258 CRISPR/Cas9 125, 331, 574, 768 Gene Correction/Modification/Targeting 18, 44, 765 Gene Expression 681 HBV, AAV 260 HCV, AAV 258 Hematopoietic Stem Cells 333, 429, 687, 767, 768 HIV 44 HIV Latency 125 HIV Stem Cell Gene Therapy 333 HIV-1, Virual Escape 331 Immunotherapy 264, 426, 428, 433, 555, 685, 699, 768 Insertional Mutagenesis 681 Lentivirus Vectors 331, 333, 431, 574 Long Term Expression 260, 429 Non-Viral Gene Delivery 173, 264, 433, 435 Non-Viral Vectorology 479 Oligonucleotide Based Therapies 440, 586 Passive Immunization 699 Pneumonia, Sepsis, Lung Contusion 173 Respiratory/Lung Disorder 22 Retrovirus Vectors 687 RNA Viral Vectors 681 RNAi and shRNA 145, 258, 260 Synthetic Gene Delivery Systems 428 Targeted Gene Expression 125 Vaccines 22, 435, 440, 479 Vector Immunology/Host Responses 79, 437 Vector Production 539 Zinc-Finger Nucleases 765 Molecular Therapy Volume 24, Supplement 1, May 2016 Copyright © The American Society of Gene & Cell Therapy
Inflammation Gene Correction/Modification/Targeting Gene Expression Genetic Diseases Lentivirus Vectors MicroRNA Oligonucleotide Based Therapies
226 146 226 226 146 146
Inflammatory Toxicity Cancer - T-Cell Therapy 197 Immunotherapy 197 Injectable Biologic Animal Models Neurological Disorders Targeted Gene Expression Insertional Mutagenesis Animal Models Gene Expression Infectious Diseases Lentivirus Vectors RNA Viral Vectors Vector Characterization/Biology
26 26 26 2 681 681 2 681 2
Insulin Therapy AAV Vectors 550 Hemophilia 550 Pharmacology/Toxicology 550 Integrase Defective Lentivirus Quantification Cancer - Cell Therapy 469 Immunotherapy 469 Lentivirus Vectors 469 Integration sites Clinical Gene Therapy Lentivirus Vectors Retrovirus Vectors
531 531 531
Intrathecal Delivery AAV Vectors Neurological Disorders
724 724
Iron-Overload AAV Vectors MicroRNA
220 220
Keloid Adenovirus Vectors Clinical Gene Therapy Gene Expression
112 112 112
Krabbe Disease AAV Vectors Genetic Diseases Neurological Disorders
57 57 57
Lentiviral Vectors AAV Vectors 322 Cancer - T-Cell Therapy 195 Chimeric Antigen Receptors 195 CRISPR/Cas9 322
S337
KEYWORD INDEX Immunotherapy 195 Musculo-Skeletal Disorders 322 Lentivirus Vector Cell Therapy - ES Cells and iPS Cells Cell Therapy - Hematopoietic Long Term Expression
234 234 234
Lentivirus Vectors AAV Vectors 129, 305, 632, 728 Adrenoleukodystrophy 250 Animal Models 2, 431, 631, 674, 756 Bone Defect 631 Cancer - Cell Therapy 212, 462, 469, 476 Cancer - Gene Therapy 214, 217, 475, 703 Cancer - Targeted Therapy 70, 217, 647, 674 Cancer - T-Cell Therapy 121, 404, 638, 648 Cell Processing 707 Cell Reprogramming 704 Cell Therapy - ES Cells and iPS Cells 241 Cell Therapy - Hematopoietic 283, 766 Cell Therapy - Mesenchymal 632 Cell Therapy - Muscle 240 Chemical Conjugates 225 Chimeric Antigen Receptors 407 Clinical Gene Therapy 250, 279, 364, 531, 534, 711, 754 CRISPR/Cas9 3, 126, 134, 135, 327, 331, 404, 532, 564, 574 Cystic Fibrosis 711 Design of Experiment 708 Gen Expression 704 Gene Correction/Modification/Targeting 3, 121, 126, 127, 129, 130, 134, 226, 286, 533, 538, 566, 682, 764 Gene Expression 5, 609, 728, 766 Genetic Diseases 226, 291, 534, 631 Genomic Deletions 135 Hematologic Diseases 4, 221, 228, 229, 230, 279, 281, 407, 682, 686, 690, 759 Hematopoietic Stem Cells 4, 135, 221, 222, 228, 229, 281, 283, 291, 333, 364, 458, 530, 536, 564, 754, 759 Hemophilia 5, 756 HIV Stem Cell Gene Therapy 333 HIV-1, Virual Escape 331 Immunotherapy 51, 225, 469, 476, 638, 647, 648 Induced Pluripotent Stem Cells 241 Infectious Diseases 331, 333, 431, 574 Inflammation 226 Insertional Mutagenesis 2 Integrase Defective Lentivirus Quantification 469 Integration Sites 531 Long Term Expression 305, 609 Macrophages, HSC 766 Mesothelioma 217 MicroRNA 589 Musculo-Skeletal Disorders 502 Nanobiotechnology 536 Neurological Disorders 250, 327 Non-Viral Vectorology 533 Ophthalmic Diseases 33 Osteoporosis, Alpha-1 Antitrypsin 632 Pharmacology/Toxicology 476, 690 Regulatory Issues 464 Respiratory/Lung Disorders 212 S338
Retrovirus Vectors 530, 531 RNA Delivery 538 RNA Viral Vectors 288, 462, 703 RNAi and shRNA 70, 532, 589 Somatic-Transgenic Technology 305 TALENs 686 Targeted Gene Expression 214 T-Cells 225 Tissue Engineering 240, 502 Toxicology studies 33, 475 Vector Characterization/Biology 2, 230, 457, 709 Vector Immunology/Host Responses 51, 764 Vector Production 286, 288, 457, 458, 464, 703, 704, 707, 708, 709, 711 Viral Transduction Enhancers 536 Zinc-Finger Nucleases 127, 130, 566 β-Thalassemias, Hemoglobinopathies 222 Lentivirus, DMD Cell Therapy - ES Cells and iPS Cells 565 Cell Therapy - Muscle 565 TALENs 565 Leukemia and Lymphoma Cancer - T-Cell Therapy Chimeric Antigen Receptors
69, 210 69, 210
Liver Diseases Animal Models Genetic Diseases Respiratory/Lung Disorders
183 183 183
Liver AAV Vectors 120 CRISPR/Cas9 120 Gene Correction/Modification/Targeting 120 Long Term Expression AAV Vectors 24, 50, 60, 305, 390, 424 Adenovirus Vectors 324, 598 Animal Models 50, 693 Antibody Delivery 429 Butyrylcholinesterase 60 Cancer - Gene Therapy 598 Cardiovascular Diseases 172 Cell Therapy - ES Cells and iPS Cells 234 Cell Therapy - Hematopoietic 234, 689 Cell Therapy - Other 161 Clinical Gene Therapy 24 Diabetes 50 Gene Expression 60, 172, 324, 609 Genetic Diseases 161, 439 HBV, AAV 260 Hematopoietic Stem Cells 429, 689 Human Immune Response to AAV 24 Immunotherapy 693 Infectious Diseases 260, 429 Lentivirus Vector 234 Lentivirus Vectors 305, 609 Neuromuscular Disorders 390 Non-Viral Gene Delivery 439 RNAi and shRNA 260 Somatic-Transgenic Technology 305 Molecular Therapy Volume 24, Supplement 1, May 2016 Copyright © The American Society of Gene & Cell Therapy
KEYWORD INDEX T-Cell Populations Vector Immunology/Host Responses Luekocyte Adhesion Deficiency AAV Vectors Cell Therapy - Hematopoietic Hematopoietic Stem Cells
693 424
Clinical Gene Therapy Lentivirus Vectors
661 217
285 285 285
Metabolic Disease AAV Vectors Animal Models Neurological Disorders
356 356 356
Lung Cancer Nanobiotechnology 489 Non-Viral Gene Delivery 489 RNAi and shRNA 489 Lysosomal Storage Deseases, Enzyme Replacement Animal Models Cell Therapy - Other Metabolic Diseases
430 430 430
Lysosomal Storage Disease AAV Vectors Genetic Diseases Neurological Disorders
348 348 348
Lysosomal Storage Diseases AAV Vectors Animal Models Neurological Disorders
361 361 361
Macrophages, HSC Cell Therapy - Hematopoietic Gene Expression Lentivirus Vectors
766 766 766
Magnetic Targeting, MRI Cell Therapy - Other MicroRNA Non-Viral Gene Delivery
338 338 338
Mathematical Modeling Clinical Gene Therapy Hematopoietic Stem Cells
314 314
Measles Virus, Glioblastoma Cancer - Gene Therapy MicroRNA RNA Viral Vectors
62 62 62
Meganuclease, AAV Vectors Cancer - T-Cell Therapy Chimeric Antigen Receptors Gene Correction/Modification/Targeting
394 394 394
Meganucleases Cancer - T-Cell Therapy Chimeric Antigen Receptors Gene Correction/Modification/Targeting Genetic Diseases Neuromuscular Disorders
200 200 138, 579 579 138
Mesothelioma Adenovirus Vectors 211, 661 Apoptosis 211 Cancer - Gene Therapy 217 Cancer - Targeted Therapy 211, 217, 661 Molecular Therapy Volume 24, Supplement 1, May 2016 Copyright © The American Society of Gene & Cell Therapy
Metabolic Diseases AAV 25 AAV Vectors 48, 58, 86, 93, 157, 158, 159, 162, 168, 218, 346, 349, 351, 352, 359, 366, 372, 382, 468, 611, 696 Adenovirus Vectors 47, 179, 484 Animal Models 166, 168, 346, 357, 430, 477 Cardiovascular Diseases 179, 358 Cell Therapy - ES Cells and iPS Cells 347 Cell Therapy - Hematopoietic 163 Cell Therapy - Mesenchymal 354 Cell Therapy - Other 430 Clinical Gene Therapy 353 Diabetes 354 Gastrointestinal Inflammation 477 Gene Correction/Modification/Targeting 48, 86, 93, 347, 354, 359 Gene Expression 158, 159, 477 Genetic Diseases 25, 46, 47, 160, 163, 166, 169, 353, 357, 358, 360, 369 Lysosomal Storage Deseases, Enzyme Replacement 430 Methylmalonic Acidemia 168 MitoTimer 358 MPS1 93 mRNA 170 Musculo-Skeletal Disorders 382 Neurological Disorders 25, 58, 157, 160, 162, 349, 351, 352, 360, 366, 369, 372, 611 Non-Viral Gene Delivery 169, 355 Ophthalmic Diseases 46 Promoterless Vector 48 RNA Delivery 170 SERCaMP, ATV6, ER Stress 218 Synthetic Gene Delivery Systems 170 TLR9 Signaling 696 Toxicology Studies 468 Urea Cycle Disorder 347 Vector Characterization/Biology 218, 355 Vector Immunology/Host Responses 696 Zinc-Finger Nucleases 484 Methylmalonic Acidemia AAV Vectors Animal Models Metabolic Diseases
168 168 168
MicroRNA AAV Vectors 220, 379, 606, 669, 737, 739 Amyotrophic Lateral Sclerosis 606 Animal Models 529, 738 Cancer - Gene Therapy 62, 144, 154, 587, 669 Cancer - Targeted Therapy 67 Cardiovascular Diseases 19 Cell Processing 245 Cell Therapy - Mesenchymal 245 S339
KEYWORD INDEX Cell Therapy - Other 338 Clinical Gene Therapy 282 Familial Hypercholesterolaemia 19 Gene Correction/Modification/Targeting 742 Gene Expression 146 Genetic Diseases 606 HCV, AAV 143 Inflammation 146 Iron-Overload 220 Lentivirus Vectors 589 Magnetic Targeting, MRI 338 Measles Virus, Glioblastoma 62 Musculo-Skeletal Disorders 379 Neurological Disorders 738, 742 Non-Viral Gene Delivery 19, 338 Oligonucleotide Based Therapies 146 Oncolytic Viruses 67 Ophthalmic Diseases 739 Prostate Cancer 669 Regulatory Issues 143 RNA Viral Vectors 62, 67, 529 RNAi and shRNA 143, 144, 154, 587, 589, 737 Targeted Gene Expression 282
Muscular Dystrophy AAV Vectors Gene Expression Musculo-Skeletal Disorders
726 726 726
93 93 93
Musculo-Skeletal Disorders AAV 499 AAV Vectors 49, 322, 378, 379, 380, 381, 382, 387, 480, 483, 497, 503, 552, 622, 624, 629, 630, 722, 726 Adenovirus Vectors 104 Animal Model 629 Animal Models 388, 499, 503, 633, 635 Cancer - Cell Therapy 636 Cell Reprogramming 525 Cell Therapy - ES Cells and iPS Cells 312 Cell Therapy - Mesenchymal 238 Cell Therapy - Muscle 238, 446, 449, 525, 577, 635, 636 Cell Therapy - Other 446, 454 Clinical Gene Therapy 328, 387, 480, 497 CRISPR/Cas9 104, 139, 317, 322, 385, 501, 504, 577, 735 DNA Viral Vectors 630 Epigenetic regulation 385 Exon Skipping 624 FSHD 153 Gene Correction/Modification/Targeting 139, 312, 317, 483, 501, 504 Gene Expression 449, 454, 499, 622, 726 Genetic Diseases 49, 381, 741 Induced Pluripotent Stem Cells 735 Lentiviral Vectors 322 Lentivirus Vectors 502 Metabolic Diseases 382 MicroRNA 379 Muscular Dystrophy 726 Neurological Disorders 741 Neuromuscular Disorders 380, 388, 500, 552, 629, 633 Non-Invasive 633 Oligonucleotide Based Therapies 153, 328, 741 Pompe Disease 49, 381 RNAi and shRNA 386, 500, 625 Spinal Muscular Atrophy, Type 1 480 Sporadic Inclusion Body Myositis, Follistatin 497 TALENs 139 Targeted Gene Expression 153 Tissue Engineering 502 Toxicology Studies 386, 625 Vaccines 722 Vector Immunology/Host Responses 378
mRNA Metabolic Diseases RNA Delivery Synthetic Gene Delivery Systems
170 170 170
Mutated Neoantigens Cancer - T-Cell Therapy 391 Gene Correction/Modification/Targeting 391 Immunotherapy 391
Mucopolysaccharidosis AAV Vectors Gene Correction/Modification/Targeting Zinc-Finger Nucleases
485 485 485
Multiple Myeloma Cancer - Gene Therapy Cancer - T-Cell Therapy Zinc-Finger Nucleases
752 752 752
Minicircle AAV Vectors DNA Viral Vectors Gene Expression Non-Viral Vectorology Ophthalmic Diseases Respiratory/Lung Disorders
306 306 743 743 306 743
Mitochondrial biogenesis Cancer - Targeted Therapy Hematologic Diseases
65 65
MitoTimer Cardiovascular Diseases Genetic Diseases Metabolic Diseases
358 358 358
Mixed Chimerism Hematologic Diseases Hematopoietic Stem Cells
231 231
MPS1 AAV Vectors Gene Correction/Modification/Targeting Metabolic Diseases
S340
Nanobiotechnology Cancer - Cell Therapy Cancer - Targeted Therapy Cancer - T-Cell Therapy Cell Delivery Chemical Conjugates Ethical Issues Gene Correction/Modification/Targeting
209, 442 209, 396, 494 396 442 342 720 563, 720
Molecular Therapy Volume 24, Supplement 1, May 2016 Copyright © The American Society of Gene & Cell Therapy
KEYWORD INDEX Hematopoietic Stem Cells 536 Lentivirus Vectors 536 Lung Cancer 489 Non-Viral Gene Delivery 339, 489, 494 Ophthalmic Diseases 339, 342 Polymeric, PBAE 494 Regulatory Issues 600 RNAi and shRNA 489 Stem Cells; Ovarian Cancer; Drug Delivery 209 Targeted Gene Therapy 339 Vaccines 600 Viral Transduction Enhancers 536 Zinc-Finger Nucleases 563 Natural History and Trial Development AAV Vectors Clinical Gene Therapy Neuromuscular Disorders
715 715 715
Neural Stem Cells, Pediatric Brain Tumors Cancer - Targeted Therapy Cell Therapy - Neuronal
443 443
Neuritin AAV Vectors Neurological Disorders
371 371
Neurological Disorders AAV 25 AAV Vectors 27, 30, 45, 54, 55, 56, 57, 58, 59, 157, 162, 186, 252, 255, 256, 271, 295, 301, 321, 337, 348, 349, 351, 352, 356, 361, 362, 366, 368, 370, 371, 372, 376, 540, 583, 604, 605, 610, 611, 612, 613, 616, 618, 619, 621, 724 Adrenoleukodystrophy 250 Aging 619 Alzheimer’s Disease 59, 567 Animal Models 26, 54, 55, 56, 186, 256, 337, 356, 361, 362, 376, 604, 612, 613, 619, 627, 738 Axon Regeneration 271 Brain Delivery 618 Cancer - Cell Therapy 528 Cancer - Gene Therapy 184 Cancer - Targeted Therapy 665 Capsid engineering 252 Cell Therapy - ES Cells and iPS Cells 528 Cell Therapy - Hematopoietic 374 Chronic Pain 111 Clinical Gene Therapy 250, 267, 367 CRISPR/Cas9 321, 327, 567 Down Syndrome 53 Enteric Nervous System, Gastrointestinal Disorders 612 Ethical Issues 627 Gene Correction/Modification/Targeting 295, 567, 605, 742 Gene Expression 252, 370, 373, 610, 616, 618, 736 Genetic Diseases 25, 27, 45, 53, 57, 160, 348, 360, 363, 369, 373, 621, 741 Glioma 665 GM2 Gangliosidosis 363 HSV Vectors 111, 114, 184, 657, 665 Injectable Biologic 26 Intrathecal Delivery 724 Krabbe Disease 57 Molecular Therapy Volume 24, Supplement 1, May 2016 Copyright © The American Society of Gene & Cell Therapy
Lentivirus Vectors 250, 327 Lysosomal Storage Disease 348 Lysosomal Storage Diseases 361 Metabolic Disease 356 Metabolic Diseases 25, 58, 157, 160, 162, 349, 351, 352, 360, 366, 369, 372, 611 MicroRNA 738, 742 Musculo-Skeletal Disorders 741 Neuritin 371 Neuromuscular Disorders 259 Neurotrophic Factor, Parkinson 610 Novel Capsid 256 Oligonucleotide Based Therapies 259, 741 Ophthalmic Diseases 267, 736 Optogenetics 186 Pharmacology/Toxicology 30, 368 Pitx3, Mitochondria, Antioxidants, Transcription 376 Retrovirus Vectors 367, 657 RNA Viral Vectors 374 RNAi and shRNA 53, 583, 614 Synthetic Gene Delivery Systems 614 Targeted Gene Expression 26, 111, 114 U1 Adaptors, Parkinsons, Huntingtons 259 Urea Cycle Disorder 45 Vector Characterization/Biology 255, 301, 540 Neuromuscular Disorders AAV Vectors 85, 90, 377, 380, 389, 390, 498, 551, 552, 629, 715, 721 Animal Model 629 Animal Models 377, 388, 498, 623, 633, 721 Cell Delivery 584 Cell Therapy - ES Cells and iPS Cells 730 Cell Therapy - Muscle 85 Clinical Gene Therapy 715 CRISPR/Cas9 730 DMD Dogs 721 Duchenne Muscular Dystrophy; Exon Skipping 584 Evolution and Development 389 Gene Correction/Modification/Targeting 138, 730 Genetic Diseases 150, 551 Long Term Expression 390 Meganucleases 138 Musculo-Skeletal Disorders 380, 388, 500, 552, 629, 633 Natural History and Trial Development 715 Neurological Disorders 259 Non-Invasive 633 Oligonucleotide Based Therapies 150, 259, 584, 623 RNAi and shRNA 500 U1 Adaptors, Parkinsons, Huntingtons 259 Vector Characterization/Biology 389 Vector Immunology/Host Responses 90 Neurotrophic Factor, Parkinson AAV Vectors Gene Expression Neurological Disorders
610 610 610
Non-Invasive Animal Models Musculo-Skeletal Disorders Neuromuscular Disorders
633 633 633 S341
KEYWORD INDEX Non-Small Cell Lung Cancer Cancer - Gene Therapy Cell Therapy - Lung RNAi and shRNA
585 585 585
Non-Viral Gene Delivery Animal Models 173, 490, 492 Bioprocess 343 Cancer - Gene Therapy 152, 155, 493, 495, 593, 599, 644, 680 Cancer - Targeted Therapy 72, 155, 494 Cancer - T-Cell Therapy 278 Cardiovascular Diseases 19 Cd19, Sleeping Beauty 278 Cell Processing 343 Cell Reprogramming 343, 344 Cell Therapy - ES Cells and iPS Cells 628 Cell Therapy - Muscle 628 Cell Therapy - Other 338 CGD Stem Cell Gene Correction 41 Chimeric Antigen Receptors 705 Clinical Gene Therapy 72 CRISPR/Cas9 41, 335, 487 DNA Vaccines 436 Exosomes 341 Familial Hypercholesterolaemia 19 Gene Correction/Modification/Targeting 41, 487, 601 Genetic Diseases 169, 439, 597, 601 Hemophilia 492 HSV Vectors 680 Immunotherapy 264, 278, 433, 495, 644 Infectious Diseases 173, 264, 433, 435 Long Term Expression 439 Lung Cancer 489 Magnetic Targeting, MRI 338 Metabolic Diseases 169, 355 MicroRNA 19, 338 Nanobiotechnology 339, 489, 494 Non-Viral Vectorology 599 Ophthalmic Diseases 339 PDGF, Angiogenesis 152 Physcial Delviery 491 Pneumonia, Sepsis, Lung Contusion 173 Polymeric, PBAE 494 Polymers 493 Respiratory/Lung Disorders 490, 597 RNA Delivery 335, 341, 593 RNAi and shRNA 152, 489, 599 Synthetic Gene Delivery Systems 493 Targeted Gene Therapy 339 Tissue Engineering 491 Transposon 628, 705 Ultrasound 492 Vaccines 435, 436, 491 Vector Characterization/Biology 344, 355 Non-Viral Vectorology Cancer - Gene Therapy Cancer - T-Cell Therapy Gene Correction/Modification/Targeting Gene Expression Hematologic Diseases S342
599 213 533 289, 743 213
Infectious Diseases 479 Lentivirus Vectors 533 Minicircle 743 Non-Viral Gene Delivery 599 Respiratory/Lung Disorders 743 RNAi and shRNA 599 Vaccines 479 Vector Characterization/Biology 289 Notch Signaling Animal Models Cardiovascular Diseases Cell Therapy - Other
447 447 447
Novel Capsid AAV Vectors Animal Models Neurological Disorders
256 256 256
Novel Molecules to Enhance Viral Entry Adenovirus Vectors 176 Pharmacology/Toxicology 176 Respiratory/Lung Disorders 176 Oligonucleotide Based Therapies Animal Models 580, 623, 740 Antisense Oligonucleotides 586 Aptamers 148, 744 Bioinformatics 148 Cancer - Cell Therapy 263 Cancer - Gene Therapy 261 Cancer - Targeted Therapy 262, 590 Cell Delivery 584 Clinical Gene Therapy 328 Duchenne Muscular Dystrophy; Exon Skipping 584 FSHD 153 Gene Expression 146 Genetic Diseases 150, 581, 741 Hematologic Diseases 581 Immunotherapy 261, 740 Infectious Diseases 440, 586 Inflammation 146 MicroRNA 146 Musculo-Skeletal Disorders 153, 328, 741 Neurological Disorders 259, 741 Neuromuscular Disorders 150, 259, 584, 623 QC/QA Assays 263 Respiratory/Lung Disorders 580 RNA Delivery 148, 262, 744 Splice, X-Linked Agammaglobulinemia 581 Synthetic Gene Delivery Systems 590 Targeted Gene Expression 153 U1 Adaptors, iRGD, KRAS, MYC, Pancreatic Cancer 262 U1 Adaptors, Parkinsons, Huntingtons 259 Vaccines 440 Oncolytic Adenovirus Cancer - Cell Therapy Cancer - T-Cell Therapy Chimeric Antigen Receptors
515 515 515
Molecular Therapy Volume 24, Supplement 1, May 2016 Copyright © The American Society of Gene & Cell Therapy
KEYWORD INDEX Oncolytic Vectors Cancer - Gene Therapy DNA Viral Vectors Hsv Vectors
520 520 520
Oncolytic Virus Animal Models 411 Apoptosis 411 Cancer - Targeted Therapy 411 Cancer - T-Cell Therapy 68 RNA Viral Vectors 68 Vaccines 68 Oncolytic Viruses Cancer - Targeted Therapy HSV Vectors Immunotherapy MicroRNA RNA Viral Vectors
67, 423, 654, 666 666 423, 666 67 67, 423, 654
Ophthalmic Diseases AAV Vectors 28, 29, 31, 80, 83, 88, 96, 98, 188, 189, 248, 249, 265, 268, 270, 306, 544, 553, 556, 607, 608, 617, 620, 739 Age-Related Macular Degeneration 189 Animal Models 96, 189 Anti-Transgene 694 Cell Therapy - Other 445 Cellular Clearance 187 Chemical Conjugates 342 Clinical Gene Therapy 83, 248, 249, 267 CRISPR/Cas9 124, 329 Delivery Techniques 29 DNA Viral Vectors 306 Exosomes 594 Functional MRI (fMRI), Visual Pathways, Brain 265 Gene Correction/Modification/Targeting 124, 188, 329, 620 Gene Expression 98, 187, 270, 320, 608, 736 Genetic Diseases 28, 46, 83, 265, 617 Glaucoma 188 Good Manufacturing Procedures 445 Induced Pluripotent Stem Cells 553, 556 Lentivirus Vectors 33 Metabolic Diseases 46 MicroRNA 739 Minicircle 306 Nanobiotechnology 339, 342 Neurological Disorders 267, 736 Non-Viral Gene Delivery 339 Optogenetic Therapy 270 Pharmacology/Toxicology 31, 88, 268 Retina 544 Targeted Gene Expression 29, 320 Targeted Gene Therapy 339 Toxicology Studies 33 Vector Characterization/Biology 594, 607 Vector Immunology/Host Responses 80, 694 Vector Transduction 594 Wet Age-Related Macular Degeneration 248
Molecular Therapy Volume 24, Supplement 1, May 2016 Copyright © The American Society of Gene & Cell Therapy
Optogenetic Therapy AAV Vectors Gene Expression Ophthalmic Diseases
270 270 270
Optogenetics AAV Vectors Animal Models Neurological Disorders
186 186 186
Orbital Shaken Bioreactors AAV Vectors Good Manufacturing Procedures Vector Production
463 463 463
Osteoporosis, Alpha-1 Antitrypsin AAV Vectors Cell Therapy - Mesenchymal Lentivirus Vectors
632 632 632
Oversized Vector AAV Vectors 92 Hemophilia 92 Vector Production 92 Pancreatic Cancer Adenovirus Vectors 639 Cancer - Targeted Therapy 639 Immunotherapy 639 Passive Immunization AAV Vectors 699 Immunotherapy 699 Infectious Diseases 699 PD-1 Cancer - T-Cell Therapy 572 CRISPR/Cas9 572 Gene Correction/Modification/Targeting 572 PDGF, Angiogenesis Cancer - Gene Therapy Non-Viral Gene Delivery RNAi and shRNA
152 152 152
Pediatric Cancers - Solid Tumors Cancer - T-Cell Therapy 649 Chimeric Antigen Receptors 649 Immunotherapy 649 Pharmacology/Toxicology AAV Vectors 30, 31, 88, 268, 299, 365, 368, 472, 550 Adenovirus Vectors 176 Aptamers 671 Cancer - Cell Therapy 476 Cancer - Targeted Therapy 671 Cell Processing 375 Cell Therapy - Neuronal 375 Gene Expression 299 GPCR, TDP-43, Cannabinoids 375 Hematologic Diseases 690 Hemophilia 550 Immunotherapy 472, 476 Insulin Therapy 550 S343
KEYWORD INDEX Lentivirus Vectors 476, 690 Neurological Disorders 30, 368 Novel Molecules to Enhance Viral Entry 176 Ophthalmic Diseases 31, 88, 268 Re-Administration 472 Respiratory/Lung Disorders 176 Toxicology Studies 365 Physcial Delviery Non-Viral Gene Delivery 491 Tissue Engineering 491 Vaccines 491 Pitx3, Mitochondria, Antioxidants, Transcription AAV Vectors Animal Models Neurological Disorders
376 376 376
Platelets Cancer - Gene Therapy Hematopoietic Stem Cells Targeted Gene Expression
668 668 668
Pneumonia, Sepsis, Lung Contusion Animal Models Infectious Diseases Non-Viral Gene Delivery
173 173 173
Polymeric, PBAE Cancer - Targeted Therapy 494 Nanobiotechnology 494 Non-Viral Gene Delivery 494 Polymers Cancer - Gene Therapy Non-Viral Gene Delivery Synthetic Gene Delivery Systems Pompe Disease AAV Vectors Genetic Diseases Musculo-Skeletal Disorders
493 493 493 49, 381 49, 381 49, 381
Precision Medicine AAV Vectors 7 Bioinformatics 537 Clinical Gene Therapy 537 CRISPR/Cas9 141, 575 Gene Correction/Modification/Targeting 575 QC/QA Assays 141 RNA Viral Vectors 537 Single-Molecule, Real-Time Sequencing 7 Vector Characterization/Biology 7 Pre-Existing AAV Antibodies AAV Vectors Animal Models Vector Immunology/Host Responses
698 698 698
Process Modeling; Monte Carlo Simulations Cancer - T-Cell Therapy Cell Processing Chimeric Antigen Receptors
650 650 650
S344
Promoterless Vector AAV Vectors Gene Correction/Modification/Targeting Metabolic Diseases
48 48 48
Promoters AAV Vectors Cell Therapy - Neuronal Gene Expression
99 99 99
Prostate Cancer AAV Vectors Cancer - Gene Therapy MicroRNA
669 669 669
Pulmonary Macrophage Transplantation Cell Therapy - Lung Cell Therapy - Other Respiratory/Lung Disorders
35 35 35
QC/QA Assays AAV Vectors 34, 290, 467 Adenovirus Vectors 461 Cancer - Cell Therapy 263 Cancer - Gene Therapy 461 Cardiovascular Diseases 244 Cell Therapy - ES Cells and iPS Cells 244 CRISPR/Cas9 141 Crude lysate 290 Hematologic Diseases 473 Oligonucleotide Based Therapies 263 Precision Medicine 141 Thermostability 34 Vector Characterization/Biology 34, 156, 473 Vector Immunology/Host Responses 467 Vector Production 156, 290 Reactive Oxygen Species Cancer - Cell Therapy Cancer - Targeted Therapy Gene Expression
216 216 216
Re-Administration AAV Vectors 472 Immunotherapy 472 Pharmacology/Toxicology 472 Regulation AAV Vectors Gene Expression Vector Characterization/Biology
546 546 546
Regulatory Issues Cell Therapy - Other 718 Clinical Gene Therapy 719 Commercialization 718 Common Rule 717 Ethical Issues 717 HCV, AAV 143 Lentivirus Vectors 464 MicroRNA 143 Nanobiotechnology 600 Regulatory Issues 719 Molecular Therapy Volume 24, Supplement 1, May 2016 Copyright © The American Society of Gene & Cell Therapy
KEYWORD INDEX RNAi and shRNA Vaccines Vector Production
143 465, 600 464, 465
Regulatory T Cell Chimeric Antigen Receptors 434 Hemophilia 434 Immunotherapy 434 Reovirus Animal Models Cancer - Targeted Therapy Vector Immunology/Host Responses
415 415 415
Respiratory/Lung Disorder Adenovirus Vectors 22 Infectious Diseases 22 Vaccines 22 Respiratory/Lung Disorders Adenovirus Vectors 176 Animal Models 167, 182, 183, 490, 580 Cancer - Cell Therapy 212 Cell Therapy - ES Cells and iPS Cells 122 Cell Therapy - Lung 35, 182 Cell Therapy - Other 35 CRISPR/Cas9 23, 167 Gene Correction/Modification/Targeting 122 Gene Expression 743 Genetic Diseases 183, 597 Lentivirus Vectors 212 Liver Diseases 183 Minicircle 743 Non-Viral Gene Delivery 490, 597 Non-Viral Vectorology 743 Novel Molecules to Enhance Viral Entry 176 Oligonucleotide Based Therapies 580 Pharmacology/Toxicology 176 Pulmonary Macrophage Transplantation 35 RNAi and shRNA 23 Targeted Gene Expression 181 Vector Characterization/Biology 181 Retina AAV Vectors Ophthalmic Diseases
544 544
Retroviral Replicating Vector Cancer - Gene Therapy 61 Clinical Gene Therapy 61 Immunotherapy 61 Retrovirus Vectors Animal Models Cancer - Gene Therapy Chemical Conjugates Clinical Gene Therapy Gene Correction/Modification/Targeting Genetic Diseases Hematopoietic Stem Cells Hsv Vectors Infectious Diseases Integration Sites Molecular Therapy Volume 24, Supplement 1, May 2016 Copyright © The American Society of Gene & Cell Therapy
284 403, 602 602 224, 367, 531 284 224 530, 687 657 687 531
Lentivirus Vectors Neurological Disorders Vector Immunology/Host Responses
530, 531 367, 657 403
RNA Delivery Apoptosis 149 Aptamers 148, 744 Bioinformatics 148 Cancer - Gene Therapy 149, 593 Cancer - Targeted Therapy 262 CRISPR/Cas9 335 Exosomes 341 Gene Correction/Modification/Targeting 538 Lentivirus Vectors 538 Metabolic Diseases 170 mRNA 170 Non-Viral Gene Delivery 335, 341, 593 Oligonucleotide Based Therapies 148, 262, 744 Sendai Virus 149 Synthetic Gene Delivery Systems 170 U1 Adaptors, iRGD, KRAS, MYC, Pancreatic Cancer 262 RNA Viral Vectors Animal Models 205, 529, 673 Bioinformatics 537 Cancer - Cell Therapy 462 Cancer - Gene Therapy 62, 201, 205, 409, 416, 506, 673, 703 Cancer - Targeted Therapy 67, 416, 423, 654 Cancer - T-Cell Therapy 68, 506 Cell Therapy - Hematopoietic 6, 374, 683 Cell Therapy - Lung 438 Clinical Gene Therapy 537 Foamy Virus 1, 683 Gene Expression 681 Hematologic Diseases 683 Hematopoietic Stem Cells 1 Immunotherapy 201, 423 In Vivo T Cell Engineering 506 Infectious Diseases 681 Insertional Mutagenesis 681 Lentivirus Vectors 288, 462, 703 Measles Virus, Glioblastoma 62 MicroRNA 62, 67, 529 Neurological Disorders 374 Oncolytic Virus 68 Oncolytic Viruses 67, 423, 654 Precision Medicine 537 Vaccines 68 Vector Characterization/Biology 1, 409 Vector Immunology/Host Responses 438 Vector Production 6, 288, 703 RNAi and shRNA AAV Vectors 145, 583, 737 Adenovirus Vectors 105 Animal Models 145 Cancer - Gene Therapy 144, 152, 154, 585, 587, 588, 599 Cancer - Targeted Therapy 70, 105 Cell Therapy - Lung 585 Clinical Gene Therapy 258 Crispr/Cas9 23, 532 Down Syndrome 53 S345
KEYWORD INDEX Genetic Diseases 53 Hbv, AAV 260 Hcv, AAV 143, 258 Infectious Diseases 145, 258, 260 Lentivirus Vectors 70, 532, 589 Long Term Expression 260 Lung Cancer 489 MicroRNA 143, 144, 154, 587, 589, 737 Musculo-Skeletal Disorders 386, 500, 625 Nanobiotechnology 489 Neurological Disorders 53, 583, 614 Neuromuscular Disorders 500 Non-Small Cell Lung Cancer 585 Non-Viral Gene Delivery 152, 489, 599 Non-Viral Vectorology 599 Pdgf, Angiogenesis 152 Regulatory Issues 143 Respiratory/Lung Disorders 23 Synthetic Gene Delivery Systems 588, 614 Toxicology Studies 386, 625 Routes Of Administration AAV Vectors Genetic Diseases Targeted Gene Expression
300 300 300
Sendai Virus Apoptosis 149 Cancer - Gene Therapy 149 RNA Delivery 149 SERCaMP, ATV6, ER Stress AAV Vectors Metabolic Diseases Vector Characterization/Biology Single-Molecule, Real-Time Sequencing AAV Vectors Precision Medicine Vector Characterization/Biology
218 218 218 7 7 7
Sleeping Beauty Transposition ; Minicircle Cancer - T-Cell Therapy 73 Chimeric Antigen Receptors 73 Immunotherapy 73 Somatic-Transgenic Technology AAV Vectors Lentivirus Vectors Long Term Expression
305 305 305
Spinal Muscular Atrophy, Type 1 AAV Vectors Clinical Gene Therapy Musculo-Skeletal Disorders
480 480 480
Splice, X-Linked Agammaglobulinemia Genetic Diseases Hematologic Diseases Oligonucleotide Based Therapies
581 581 581
S346
Sporadic Inclusion Body Myositis, Follistatin AAV Vectors Clinical Gene Therapy Musculo-Skeletal Disorders
497 497 497
Stem Cells; Ovarian Cancer; Drug Delivery Cancer - Cell Therapy 209 Cancer - Targeted Therapy 209 Nanobiotechnology 209 Synthetic Biology CRISPR/Cas9 136 Gene Correction/Modification/Targeting 136 TALENs 136 Synthetic Gene Delivery Systems Cancer - Gene Therapy 493, 588 Cancer - Targeted Therapy 590 Immunotherapy 428 Infectious Diseases 428 Metabolic Diseases 170 mRNA 170 Neurological Disorders 614 Non-Viral Gene Delivery 493 Oligonucleotide Based Therapies 590 Polymers 493 RNA Delivery 170 RNAi and shRNA 588, 614 TALEN Cell Therapy - Hematopoietic Gene Correction/Modification/Targeting Hematopoietic Stem Cells
562 562 562
TALENs AAV Vectors 316, 569 Animal Models 758 Cancer - T-Cell Therapy 397, 676 CD40L, X-HIGM, Gene Editing 316 Cell Therapy - ES Cells and iPS Cells 332, 565 Cell Therapy - Muscle 565 Chimeric Antigen Receptors 397, 676 CRISPR/Cas 569 CRISPR/Cas9 136, 137, 139, 692 Gene Correction/Modification/Targeting 132, 136, 137, 139, 325, 332, 569 Genetic Diseases 325 Hematologic Diseases 686, 692 Hematopoietic Stem Cells 316, 758 Immunodeficiency 325 Immunotherapy 132 Lentivirus Vectors 686 Lentivirus, DMD 565 Musculo-Skeletal Disorders 139 Synthetic Biology 136 Thalassemia 692 Tandem Bispecific Chimeric Antigen Receptors Cancer - Targeted Therapy Cancer - T-Cell Therapy Chimeric Antigen Receptors
392 392 392
Molecular Therapy Volume 24, Supplement 1, May 2016 Copyright © The American Society of Gene & Cell Therapy
KEYWORD INDEX Targeted Epigenetic Editing CRISPR/Cas9 729 Epigenetic Regulation 729 Gene Correction/Modification/Targeting 729
T-Cell Populations Animal Models 693 Immunotherapy 693 Long Term Expression 693
Targeted Gene Expression AAV Vectors 29, 298, 300, 307, 478, 542, 695, 723 Animal Models 26, 675 Barcoding 542 Cancer - Cell Therapy 679 Cancer - Gene Therapy 214, 668 Cancer - Targeted Therapy 723 Cancer - T-Cell Therapy 76 Cancer Stem Cells, Tumor Microenvironment 679 Cell Reprogramming 40 Chronic Pain 111 Clinical Gene Therapy 282 Colorectal Cancer 675 CRISPR/Cas9 125, 557, 626 Delivery Techniques 29 DNA Viral Vectors 108 FSHD 153 Gene Correction/Modification/Targeting 40, 315, 542 Gene Expression 320, 675, 679 Genetic Diseases 300 Hematopoietic Stem Cells 668 HIV Latency 125 HSV Vectors 111, 114 IL13Ra2, GBM, Solid Tumors 76 Immunotherapy 76 Induced Pluripotent Stem Cells 557, 626 Infectious Diseases 125 Injectable Biologic 26 Lentivirus Vectors 214 MicroRNA 282 Musculo-Skeletal Disorders 153 Neurological Disorders 26, 111, 114 Oligonucleotide Based Therapies 153 Ophthalmic Diseases 29, 320 Platelets 668 Respiratory/Lung Disorders 181 Routes of Administration 300 Vector Characterization/Biology 108, 181, 298, 307, 478, 695 Xist-Mediated Chromosome Silencing 315 Zinc-Finger Nucleases 315
T-Cells Chemical Conjugates 225 Immunotherapy 225 Lentivirus Vectors 225
Targeted Gene Therapy Nanobiotechnology 339 Non-Viral Gene Delivery 339 Ophthalmic Diseases 339 Targeting AAV Vectors Chimeric Antigen Receptors
251 251
T-Cell Development, Infections, Notch, Thymus Cell Processing 425 Genetic Diseases 425 Immunotherapy 425
Molecular Therapy Volume 24, Supplement 1, May 2016 Copyright © The American Society of Gene & Cell Therapy
Telomerase Cancer - Cell Therapy 276 Hematologic Diseases 276 Immunotherapy 276 Thalassemia Crispr/Cas9 692 Hematologic Diseases 692 TALENs 692 Therapeutic Gene Modulation Animal Models Hsv Vectors Thermostability AAV Vectors QC/QA Assays Vector Characterization/Biology
656 656 34 34 34
Tissue Engineering AAV Vectors 524, 702 Cardiovascular Diseases 246 Cell Delivery 340 Cell Therapy - Hematopoietic 239 Cell Therapy - Mesenchymal 246, 524 Cell Therapy - Muscle 240 Cell Therapy - Other 340, 452 Commercialisation 452 Crispr/Cas9 318 Immunotherapy 239 Induced Pluripotent Stem Cells 318 Lentivirus Vectors 240, 502 Musculo-Skeletal Disorders 502 Non-Viral Gene Delivery 491 Physcial Delviery 491 Vaccines 491 Vector Immunology/Host Responses 702 Tlr9 Signaling AAV Vectors Metabolic Diseases Vector Immunology/Host Responses
696 696 696
Toxicology Studies AAV Vectors 365, 468, 470 Cancer - Gene Therapy 475 Chimeric Antigen Receptors 399 Clinical Gene Therapy 470 Genetic Diseases 32 Hematopoietic Stem Cells 32 Immunotherapy 399 Lentivirus Vectors 33, 475 Metabolic Diseases 468 Musculo-Skeletal Disorders 386, 625 S347
KEYWORD INDEX Ophthalmic Diseases 33 Pharmacology/Toxicology 365 RNAi and shRNA 386, 625 Transgene Engineering AAV Vectors 456 Gene Expression 456 Hemophilia 456 Transposon Cell Therapy - ES Cells and iPS Cells Cell Therapy - Muscle Chimeric Antigen Receptors Non-Viral Gene Delivery
628 628 705 628, 705
Tumor Microenvironment Adenovirus Vectors 662 Cancer - Gene Therapy 662 Immunotherapy 662 Tumor-Specific Promoter Cancer - Gene Therapy Cancer - Targeted Therapy Hematologic Diseases
677 677 677
Tunneling Nanotubes Cell Delivery Cell Therapy - Hematopoietic Genetic Diseases
336 336 336
U1 Adaptors, iRGD, KRAS, MYC, Pancreatic Cancer Cancer - Targeted Therapy Oligonucleotide Based Therapies RNA Delivery Neurological Disorders Neuromuscular Disorders Oligonucleotide Based Therapies
262 262 262 259 259 259
Ultrasound Animal Models 492 Hemophilia 492 Non-Viral Gene Delivery 492 Urea Cycle Disorder AAV Vectors Cell Therapy - ES Cells and iPS Cells Gene Correction/Modification/Targeting Genetic Diseases Metabolic Diseases Neurological Disorders
45 347 347 45 347 45
Vaccines AAV Vectors 196, 722 Adenovirus Vectors 22, 36, 642 Cancer - Gene Therapy 643 Cancer - T-Cell Therapy 68, 191, 596 Cell Delivery 596 Clinical Gene Therapy 36 DNA Vaccines 436 DNA Viral Vectors 712 Immunotherapy 191, 196, 395, 642, 643 Infectious Diseases 22, 435, 440, 479 Musculo-Skeletal Disorders 722 Nanobiotechnology 600 S348
Non-Viral Gene Delivery Non-Viral Vectorology Oligonucleotide Based Therapies Oncolytic Virus Physcial Delviery Regulatory Issues Respiratory/Lung Disorder RNA Viral Vectors Tissue Engineering Vector Immunology/Host Responses Vector Production
435, 436, 491 479 440 68 491 465, 600 22 68 491 395 465, 712
Vector Characterization/Biology AAV Vectors 7, 8, 9, 10, 11, 12, 34, 82, 91, 218, 254, 255, 257, 293, 298, 301, 302, 304, 307, 389, 474, 478, 540, 541, 546, 547, 548, 607, 695, 727 Adenovirus Vectors 14, 17, 655 Animal Models 2 Cancer - Gene Therapy 409, 548 Cancer - Targeted Therapy 655 Cell Reprogramming 344 Clinical Gene Therapy 547 Crispr/Cas9 293 DNA Viral Vectors 14, 17, 107, 108, 254, 727 Epigenetic Regulation 8 Evolution and Development 389 Exosomes 594 Foamy Virus 1 Gene Expression 10, 11, 91, 289, 474, 546 Genetic Diseases 257 Hematologic Diseases 230, 473 Hematopoietic Stem Cells 1 Hsv Vectors 107 Humoral Immune Response 82 Insertional Mutagenesis 2 Lentivirus Vectors 2, 230, 457, 709 Metabolic Diseases 218, 355 Neurological Disorders 255, 301, 540 Neuromuscular Disorders 389 Non-Viral Gene Delivery 344, 355 Non-Viral Vectorology 289 Ophthalmic Diseases 594, 607 Precision Medicine 7 QC/QA Assays 34, 156, 473 Regulation 546 Respiratory/Lung Disorders 181 RNA Viral Vectors 1, 409 SERCaMP, ATV6, ER Stress 218 Single-Molecule, Real-Time Sequencing 7 Targeted Gene Expression 108, 181, 298, 307, 478, 695 Thermostability 34 Vector Immunology/Host Responses 82 Vector Production 9, 12, 156, 302, 304, 457, 709 Vector Transduction 594 Viral Evolution 541 Vector Immunology/Host Responses AAV Vectors 77, 78, 79, 80, 81, 82, 90, 185, 310, 378, 424, 467, 595, 696, 697, 698, 700, 701, 702 Adenovirus Vectors 517 Animal Models 63, 415, 437, 698 Antibody Response 77 Molecular Therapy Volume 24, Supplement 1, May 2016 Copyright © The American Society of Gene & Cell Therapy
KEYWORD INDEX Anti-Transgene 694 Cancer - Gene Therapy 403 Cancer - Targeted Therapy 208, 415, 517 CD8+ T Cells, Ovalbumin 79 Cell Therapy - Lung 438 Cell Therapy - Neuronal 208 Clinical Gene Therapy 185, 697 Exosomes 595 Gene Correction/Modification/Targeting 764 Gene Expression 310, 700, 701 Hemophilia 78 Hsv Vectors 518 Humoral Immune Response 82 Immunotherapy 51, 63, 81, 395, 518 Infectious Diseases 79, 437 Lentivirus Vectors 51, 764 Long Term Expression 424 Metabolic Diseases 696 Musculo-Skeletal Disorders 378 Neuromuscular Disorders 90 Ophthalmic Diseases 80, 694 Pre-Existing AAV Antibodies 698 QC/QA Assays 467 Reovirus 415 Retrovirus Vectors 403 RNA Viral Vectors 438 Tissue Engineering 702 Tlr9 Signaling 696 Vaccines 395 Vector Characterization/Biology 82 Vector Production AAV Vectors 9, 12, 87, 92, 94, 95, 97, 100, 101, 290, 292, 294, 302, 304, 308, 309, 460, 463, 471, 539, 549, 591, 672, 684, 710 Adenovirus Vectors 706, 713 Cancer - Gene Therapy 672, 703 Capsid Modification 95 Cell Processing 707 Cell Reprogramming 704 Cell Therapy - Hematopoietic 6 Chromatography 100 Clinical Gene Therapy 711 Continuous Flow Ultracentrifugation 97 Crude Lysate 290 Cystic Fibrosis 711 Database Development 591 Design Of Experiment 708 DNA Viral Vectors 87, 97, 294, 549, 712 Gen Expression 704 Gene Correction/Modification/Targeting 286 Gene Expression 292, 471 Gene Therapy 94 Genetic Diseases 101 Gmp Manufacturing 460, 710 Good Manufacturing Procedures 100, 308, 309, 460, 463, 591, 706, 710, 713 Hematopoietic Stem Cells 458 Hemophilia 92, 684 Hsv Vectors 94 Hybrid Vector 672 Infectious Diseases 539 Molecular Therapy Volume 24, Supplement 1, May 2016 Copyright © The American Society of Gene & Cell Therapy
Lentivirus Vectors
286, 288, 457, 458, 464, 703, 704, 707, 708, 709, 711 Orbital Shaken Bioreactors 463 Oversized Vector 92 QC/QA Assays 156, 290 Regulatory Issues 464, 465 RNA Viral Vectors 6, 288, 703 Vaccines 465, 712 Vector Characterization/Biology 9, 12, 156, 302, 304, 457, 709 Vector Purification 294 XLRP 101 Vector Purification AAV Vectors DNA Viral Vectors Vector Production
294 294 294
Vector Transduction Exosomes 594 Ophthalmic Diseases 594 Vector Characterization/Biology 594 Viral Evolution AAV Vectors Vector Characterization/Biology
541 541
Viral Transduction Enhancers Hematopoietic Stem Cells 536 Lentivirus Vectors 536 Nanobiotechnology 536 Wet Age-Related Macular Degeneration AAV Vectors Clinical Gene Therapy Ophthalmic Diseases
248 248 248
WT1 Cancer - T-Cell Therapy 193 Chimeric Antigen Receptors 193 Immunotherapy 193 XIST-Mediated Chromosome Silencing Gene Correction/Modification/Targeting Targeted Gene Expression Zinc-Finger Nucleases
315 315 315
XLRP AAV Vectors Genetic Diseases Vector Production
101 101 101
Zinc-Finger Nucleases AAV Vectors 133, 485 Adenovirus Vectors 484 Animal Models 38 Cancer - Cell Therapy 641 Cancer - Gene Therapy 752 Cancer - T-Cell Therapy 752 Cell Therapy - Hematopoietic 734 Gene Correction/Modification/Targeting 37, 38, 118, 127, 130, 133, 315, 485, 563, 566, 641, 765 Genetic Modification 734 Hematologic Diseases 118 S349
KEYWORD INDEX Hematopoietic Stem Cells 37, 734 Infectious Diseases 765 Lentivirus Vectors 127, 130, 566 Metabolic Diseases 484 Mucopolysaccharidosis 485 Multiple Myeloma 752 Nanobiotechnology 563 Targeted Gene Expression 315 XIST-Mediated Chromosome Silencing 315 β-Thalassemias, Hemoglobinopathies Hematopoietic Stem Cells Lentivirus Vectors
S350
222 222
Molecular Therapy Volume 24, Supplement 1, May 2016 Copyright © The American Society of Gene & Cell Therapy
Gene Expression 10, 11, 60, 91, 98, 99, 158, 159, 164, 252, 270, 292, 299, 310, 370, 456, 471, 474, 545, 546, 608, 610, 616, 618, 622, 700, 701, 726, 728 Gene Therapy 94 Genetic Diseases 27, 28, 45, 49, 57, 83, 101, 164, 165, 257, 265, 269, 300, 348, 350, 381, 481, 551, 606, 617, 621, 755 Glaucoma 188 GMP Manufacturing 460, 710 Good Manufacturing Procedures 100, 308, 309, 460, 463, 591, 592, 710 Hearing 269 Hematologic Diseases 227, 760 Hematopoietic Stem Cells 285, 316 Hemophilia 52, 78, 92, 223, 227, 253, 296, 456, 545, 550, 684, 688, 760 High Quality Grade 592 HIV 44 HSV Vectors 94 Human Immune Response to AAV 24 Humoral Immune Response 82 Hybrid Vector 672 Immunotherapy 81, 196, 472, 555, 688, 699 Induced Pluripotent Stem Cells 553, 556 Infectious Diseases 44, 79, 145, 539, 555, 699 Insulin Therapy 550 Intrathecal Delivery 724 Iron-Overload 220 Krabbe Disease 57 Lentiviral Vectors 322 Lentivirus Vectors 129, 305, 632, 728 Liver 120 Long Term Expression 24, 50, 60, 305, 390, 424 Luekocyte Adhesion Deficiency 285 Lysosomal Storage Disease 348 Lysosomal Storage Diseases 361 Metabolic Disease 356 Metabolic Diseases 48, 58, 86, 93, 157, 158, 159, 162, 168, 218, 346, 349, 351, 352, 359, 366, 372, 382, 468, 611, 696 Methylmalonic Acidemia 168 MicroRNA 220, 379, 606, 669, 737, 739 Minicircle 306 MPS1 93 Mucopolysaccharidosis 485 Muscular Dystrophy 726 Musculo-Skeletal Disorders 49, 322, 378, 379, 380, 381, 382, 387, 480, 483, 497, 503, 552, 622, 624, 629, 630, 722, 726 Natural History and Trial Development 715 Neuritin 371 Neurological Disorders 27, 30, 45, 54, 55, 56, 57, 58, 59, 157, 162, 186, 252, 255, 256, 271, 295, 301, 321, 337, 348, 349, 351, 352, 356, 361, 362, 366, 368, 370, 371, 372, 376, 540, 583, 604, 605, 610, 611, 612, 613, 616, 618, 619, 621, 724 Neuromuscular Disorders 85, 90, 377, 380, 389, 390, 498, 551, 552, 629, 715, 721 Neurotrophic Factor, Parkinson 610 Novel Capsid 256
S321
KEYWORD INDEX Ophthalmic Diseases 28, 29, 31, 80, 83, 88, 96, 98, 188, 189, 248, 249, 265, 268, 270, 306, 544, 553, 556, 607, 608, 617, 620, 739 Optogenetic Therapy 270 Optogenetics 186 Orbital Shaken Bioreactors 463 Osteoporosis, Alpha-1 Antitrypsin 632 Oversized Vector 92 Passive Immunization 699 Pharmacology/Toxicology 30, 31, 88, 268, 299, 365, 368, 472, 550 Pitx3, Mitochondria, Antioxidants, Transcription 376 Pompe Disease 49, 381 Precision Medicine 7 Pre-Existing AAV Antibodies 698 Promoterless Vector 48 Promoters 99 Prostate Cancer 669 QC/QA Assays 34, 290, 467 Re-Administration 472 Regulation 546 Retina 544 RNAi and shRNA 145, 583, 737 Routes of Administration 300 SERCaMP, ATV6, ER Stress 218 Single-Molecule, Real-Time Sequencing 7 Somatic-Transgenic Technology 305 Spinal Muscular Atrophy, Type 1 480 Sporadic Inclusion Body Myositis, Follistatin 497 TALENs 316, 569 Targeted Gene Expression 29, 298, 300, 307, 478, 542, 695, 723 Targeting 251 Thermostability 34 Tissue Engineering 524, 702 TLR9 Signaling 696 Toxicology Studies 365, 468, 470 Transgene Engineering 456 Urea Cycle Disorder 45 Vaccines 196, 722 Vector Characterization/Biology 7, 8, 9, 10, 11, 12, 34, 82, 91, 218, 254, 255, 257, 293, 298, 301, 302, 304, 307, 389, 474, 478, 540, 541, 546, 547, 548, 607, 695, 727 Vector Immunology/Host Responses 77, 78, 79, 80, 81, 82, 90, 185, 310, 378, 424, 467, 595, 696, 697, 698, 700, 701, 702 Vector Production 9, 12, 87, 92, 94, 95, 97, 100, 101, 290, 292, 294, 302, 304, 308, 309, 460, 463, 471, 539, 549, 591, 672, 684, 710 Vector Purification 294 Viral Evolution 541 Wet Age-Related Macular Degeneration 248 XLRP 101 Zinc-Finger Nucleases 133, 485 AAV Animal Models Gene Expression Genetic Diseases Metabolic Diseases Musculo-Skeletal Disorders Neurological Disorders S322
499 499 25 25 499 25
Adenovirus Cytokine Cancer - Gene Therapy 405 Cancer - T-Cell Therapy 405 Immunotherapy 405 Adenovirus Vectors AAV Vectors 102 Animal Models 16, 417, 519 Apoptosis 211, 659 Breast Cancer 417 Cancer - Cell Therapy 117, 420 Cancer - Gene Therapy 16, 113, 117, 413, 414, 417, 418, 421, 461, 509, 514, 516, 598, 659, 662 Cancer - Targeted Therapy 13, 103, 105, 211, 408, 421, 517, 519, 639, 655, 661 Cancer - T-Cell Therapy 410, 513 Cardiovascular Diseases 179 Cell Delivery 420 Cell Therapy - Hematopoietic 757 Cell Therapy - Mesenchymal 453 Checkpoint Inhibitor 513 Clinical Gene Therapy 36, 109, 110, 112, 115, 116, 453, 514, 661 CRISPR/Cas9 15, 104 DNA Viral Vectors 13, 14, 17, 102, 106 Gene Correction/Modification/Targeting 15, 18 Gene Expression 109, 110, 112, 113, 115, 116, 324, 413 Genetic Diseases 47 Good Manufacturing Procedures 706, 713 Hematopoietic Stem Cells 106, 757 Immunotherapy 408, 410, 414, 418, 509, 513, 516, 639, 642, 662 Infectious Diseases 18, 22, 103 Keloid 112 Long Term Expression 324, 598 Mesothelioma 211, 661 Metabolic Diseases 47, 179, 484 Musculo-Skeletal Disorders 104 Novel Molecules to Enhance Viral Entry 176 Pancreatic Cancer 639 Pharmacology/Toxicology 176 QC/QA Assays 461 Respiratory/Lung Disorder 22 Respiratory/Lung Disorders 176 RNAi and shRNA 105 Tumor Microenvironment 662 Vaccines 22, 36, 642 Vector Characterization/Biology 14, 17, 655 Vector Immunology/Host Responses 517 Vector Production 706, 713 Zinc-Finger Nucleases 484 Adrenoleukodystrophy Clinical Gene Therapy Lentivirus Vectors Neurological Disorders
250 250 250
Age-Related Macular Degeneration AAV Vectors Animal Models Ophthalmic Diseases
189 189 189
Molecular Therapy Volume 24, Supplement 1, May 2016 Copyright © The American Society of Gene & Cell Therapy
KEYWORD INDEX Aging AAV Vectors Animal Models Neurological Disorders
619 619 619
Alzheimer’s Disease AAV Vectors 59 CRISPR/Cas9 567 Gene Correction/Modification/Targeting 567 Neurological Disorders 59, 567 Amyotrophic Lateral Sclerosis AAV Vectors Genetic Diseases MicroRNA
606 606 606
Animal Model AAV Vectors Musculo-Skeletal Disorders Neuromuscular Disorders
629 629 629
Animal Models AAV 499 AAV Vectors 50, 52, 54, 55, 56, 84, 96, 145, 168, 180, 186, 189, 223, 227, 256, 296, 337, 346, 350, 356, 361, 362, 376, 377, 498, 503, 604, 612, 613, 615, 619, 698, 721, 725, 760 Adenovirus Vectors 16, 417, 519 Age-Related Macular Degeneration 189 Aging 619 Animal Models 84 Apoptosis 411 Autoimmune Disease 84 Bone Defect 631 Breast Cancer 417 Cancer - Gene Therapy 16, 205, 412, 417, 667, 673 Cancer - Targeted Therapy 411, 412, 415, 519, 660, 674 Cancer - T-Cell Therapy 275 Cardiopulmonary 383 Cardiovascular Diseases 180, 447, 448 Cell Therapy - Hematopoietic 427 Cell Therapy - Lung 182 Cell Therapy - Mesenchymal 448 Cell Therapy - Muscle 635 Cell Therapy - Other 430, 447 Chimeric Antigen Receptors 750 Clinical Gene Therapy 383, 615 Colorectal Cancer 675 CRISPR/Cas9 167 Cytokines 441 Diabetes 50 DMD Dogs 721 Enteric Nervous System, Gastrointestinal Disorders 612 Esophageal Squamous Cell Carcinoma 412 Ethical Issues 627 Gastrointestinal Inflammation 477 Gene Correction/Modification/Targeting 38, 284, 725 Gene Expression 64, 441, 477, 499, 675, 749 Genetic Diseases 166, 183, 350, 357, 383, 631 Hematologic Diseases 227, 760 Hematopoietic Stem Cells 758, 767 Hemophilia 52, 223, 227, 296, 492, 756, 760 Molecular Therapy Volume 24, Supplement 1, May 2016 Copyright © The American Society of Gene & Cell Therapy
HIV 427 HSV Vectors 656, 660, 667 Immunotherapy 63, 64, 275, 427, 693, 740, 749, 750 Infectious Diseases 145, 173, 431, 437, 767 Injectable Biologic 26 Insertional Mutagenesis 2 Lentivirus Vectors 2, 431, 631, 674, 756 Liver Diseases 183 Long Term Expression 50, 693 Lysosomal Storage Deseases, Enzyme Replacement 430 Lysosomal Storage Diseases 361 Metabolic Disease 356 Metabolic Diseases 166, 168, 346, 357, 430, 477 Methylmalonic Acidemia 168 MicroRNA 529, 738 Musculo-Skeletal Disorders 388, 499, 503, 633, 635 Neurological Disorders 26, 54, 55, 56, 186, 256, 337, 356, 361, 362, 376, 604, 612, 613, 619, 627, 738 Neuromuscular Disorders 377, 388, 498, 623, 633, 721 Non-Invasive 633 Non-Viral Gene Delivery 173, 490, 492 Notch Signaling 447 Novel Capsid 256 Oligonucleotide Based Therapies 580, 623, 740 Oncolytic Virus 411 Ophthalmic Diseases 96, 189 Optogenetics 186 Pitx3, Mitochondria, Antioxidants, Transcription 376 Pneumonia, Sepsis, Lung Contusion 173 Pre-Existing AAV Antibodies 698 Reovirus 415 Respiratory/Lung Disorders 167, 182, 183, 490, 580 Retrovirus Vectors 284 RNA Viral Vectors 205, 529, 673 RNAi and shRNA 145 TALENs 758 Targeted Gene Expression 26, 675 T-Cell Populations 693 Therapeutic Gene Modulation 656 Ultrasound 492 Vector Characterization/Biology 2 Vector Immunology/Host Responses 63, 415, 437, 698 Zinc-Finger Nucleases 38 Anti CD19 CAR T KTE-C19 Cancer - Cell Therapy Cancer - T-Cell Therapy Chimeric Antigen Receptors
745 745 745
Antibody Delivery Hematopoietic Stem Cells Infectious Diseases Long Term Expression
429 429 429
Antibody Response AAV Vectors Vector Immunology/Host Responses Antisense Oligonucleotides Infectious Diseases Oligonucleotide Based Therapies
77 77 586 586
S323
KEYWORD INDEX Anti-Transgene Ophthalmic Diseases Vector Immunology/Host Responses
694 694
Apoptosis Adenovirus Vectors 211, 659 Animal Models 411 Cancer - Gene Therapy 149, 345, 659 Cancer - Targeted Therapy 211, 411 Chemical Conjugates 345 Curcumin, CDC, Breast Cancer 345 Mesothelioma 211 Oncolytic Virus 411 RNA Delivery 149 Sendai Virus 149 Aptamers Bioinformatics 148 Cancer - Targeted Therapy 671 Oligonucleotide Based Therapies 148, 744 Pharmacology/Toxicology 671 RNA Delivery 148, 744 Assay Development Clinical Gene Therapy 171 Gene Expression 171 Hemophilia 171 Auditory Diseases AAV Vectors 269 Genetic Diseases 269 Hearing 269 Autoimmune disease AAV Vectors Animal Models
84 84
Axon Regeneration AAV Vectors Neurological Disorders
271 271
Barcoding AAV Vectors Gene Correction/Modification/Targeting Targeted Gene Expression
542 542 542
Bioinformatics Aptamers 148 Clinical Gene Therapy 537 Oligonucleotide Based Therapies 148 Precision Medicine 537 RNA Delivery 148 RNA Viral Vectors 537 Bioprocess Cell Processing Cell Reprogramming Non-Viral Gene Delivery
343 343 343
Bone Defect Animal Models Genetic Diseases Lentivirus Vectors
631 631 631
S324
Brain Delivery AAV Vectors Gene Expression Neurological Disorders
618 618 618
Breast Cancer Adenovirus Vectors Animal Models Cancer - Gene Therapy
417 417 417
Butyrylcholinesterase AAV Vectors Gene Expression Long Term Expression
60 60 60
Cancer - Cell Therapy AAV Vectors 678 Adenovirus Vectors 117, 420 Anti CD19 CAR T KTE-C19 745 Cancer - Gene Therapy 66, 117, 192, 207 Cancer - Targeted Therapy 66, 204, 209, 216, 393 Cancer - T-Cell Therapy 192, 199, 204, 393, 402, 515, 646, 652, 745, 748 Cancer Stem Cells, Tumor Microenvironment 679 Cell Delivery 242, 420, 442, 511 Cell Processing 402, 459 Cell Reprogramming 645 Cell Therapy - ES Cells and iPS cells 528 Cell Therapy - Hematopoietic 233, 273 Cell Therapy - Mesenchymal 242 Cell Therapy - Muscle 636 Cell Therapy - Neuronal 678 Cell Therapy - Other 645 Chimeric Antigen Receptor T Cells 204 Chimeric Antigen Receptors 207, 515, 745, 748 CRISPR/Cas9 422 Gene Correction/Modification/Targeting 641 Gene Expression 216, 679 Good Manufacturing Procedures 459 Hematologic Diseases 233, 276 Hematopoietic Stem Cells 273 Immunotherapy 199, 276, 422, 469, 476, 511, 646, 652 Integrase Defective Lentivirus Quantification 469 Lentivirus Vectors 212, 462, 469, 476 Musculo-Skeletal Disorders 636 Nanobiotechnology 209, 442 Neurological Disorders 528 Oligonucleotide Based Therapies 263 Oncolytic Adenovirus 515 Pharmacology/Toxicology 476 QC/QA Assays 263 Reactive Oxygen Species 216 Respiratory/Lung Disorders 212 RNA Viral Vectors 462 Stem Cells; Ovarian Cancer; Drug Delivery 209 Targeted Gene Expression 679 Telomerase 276 Zinc-Finger Nucleases 641 Cancer - Gene Therapy AAV Vectors Adenovirus Cytokine
215, 219, 323, 548, 669, 672 405 Molecular Therapy Volume 24, Supplement 1, May 2016 Copyright © The American Society of Gene & Cell Therapy
KEYWORD INDEX Adenovirus Vectors
16, 113, 117, 413, 414, 417, 418, 421, 461, 509, 514, 516, 598, 659, 662 Animal Models 16, 205, 412, 417, 667, 673 Apoptosis 149, 345, 659 Breast Cancer 417 Cancer - Cell Therapy 66, 117, 192, 207 Cancer - Targeted Therapy 66, 155, 217, 219, 412, 416, 419, 421, 658, 670, 677, 762 Cancer - T-Cell Therapy 74, 192, 206, 323, 405, 506, 653, 746, 747, 752, 762 Cell Therapy - Hematopoietic 523 Cell Therapy - Lung 585 Chemical Conjugates 345, 602 Chimeric Antigen Receptor, CAR 670 Chimeric Antigen Receptors 74, 207, 747 Clinical Gene Therapy 61, 514 Colorectal Cancer 664 Curcumin, CDC, Breast Cancer 345 Cytotoxic Transgene 215 DNA Viral Vectors 419, 520, 664 Esophageal Squamous Cell Carcinoma 412 Ex-Vivo Expansion 523 Gene Expression 113, 142, 413 Hematologic Diseases 677 Hematopoietic Stem Cells 523, 668 HSV Vectors 184, 520, 658, 664, 667, 680 Hybrid Vector 672 Immunotherapy 61, 142, 201, 206, 261, 405, 414, 418, 495, 509, 516, 643, 644, 653, 662, 746 In Vivo T Cell Engineering 506 Lentivirus Vectors 214, 217, 475, 703 Long Term Expression 598 Measles Virus, Glioblastoma 62 Mesothelioma 217 MicroRNA 62, 144, 154, 587, 669 Multiple Myeloma 752 Neurological Disorders 184 Non-Small Cell Lung Cancer 585 Non-Viral Gene Delivery 152, 155, 493, 495, 593, 599, 644, 680 Non-Viral Vectorology 599 Oligonucleotide Based Therapies 261 Oncolytic Vectors 520 PDGF, Angiogenesis 152 Platelets 668 Polymers 493 Prostate Cancer 669 QC/QA Assays 461 Retroviral Replicating Vector 61 Retrovirus Vectors 403, 602 RNA Delivery 149, 593 RNA Viral Vectors 62, 201, 205, 409, 416, 506, 673, 703 RNAi and shRNA 144, 152, 154, 585, 587, 588, 599 Sendai Virus 149 Synthetic Gene Delivery Systems 493, 588 Targeted Gene Expression 214, 668 Toxicology Studies 475 Tumor Microenvironment 662 Tumor-Specific Promoter 677 Vaccines 643 Vector Characterization/Biology 409, 548 Vector Immunology/Host Responses 403 Molecular Therapy Volume 24, Supplement 1, May 2016 Copyright © The American Society of Gene & Cell Therapy
Vector Production Zinc-Finger Nucleases
672, 703 752
Cancer - Targeted Therapy AAV Vectors 89, 219, 723 Adenovirus Vectors 13, 103, 105, 211, 408, 421, 517, 519, 639, 655, 661 Animal Models 411, 412, 415, 519, 660, 674 Apoptosis 211, 411 Aptamers 671 Cancer - Cell Therapy 66, 204, 209, 216, 393 Cancer - Gene Therapy 66, 155, 217, 219, 412, 416, 419, 421, 658, 670, 677, 762 Cancer - Targeted Therapy 670 Cancer - T-Cell Therapy 202, 204, 392, 393, 396, 505, 762 Cardiovascular Diseases 89 Cell Therapy - Neuronal 208, 443 Chimeric Antigen Receptor T Cells 204 Chimeric Antigen Receptor, CAR 670 Chimeric Antigen Receptors 392 Clinical Gene Therapy 72, 661 DNA Vaccines, Electroporation 401 DNA Viral Vectors 13, 419 Esophageal Squamous Cell Carcinoma 412 Gene Expression 216 Glioma 665 Hematologic Diseases 65, 663, 677 HSV Vectors 658, 660, 665, 666 Immunotherapy 202, 401, 408, 423, 505, 639, 647, 663, 666 Infectious Diseases 103 Lentivirus Vectors 70, 217, 647, 674 Mesothelioma 211, 217, 661 MicroRNA 67 Mitochondrial Biogenesis 65 Nanobiotechnology 209, 396, 494 Neural Stem Cells, Pediatric Brain Tumors 443 Neurological Disorders 665 Non-Viral Gene Delivery 72, 155, 494 Oligonucleotide Based Therapies 262, 590 Oncolytic Virus 411 Oncolytic Viruses 67, 423, 654, 666 Pancreatic Cancer 639 Pharmacology/Toxicology 671 Polymeric, PBAE 494 Reactive Oxygen Species 216 Reovirus 415 RNA Delivery 262 RNA Viral Vectors 67, 416, 423, 654 RNAi and shRNA 70, 105 Stem Cells; Ovarian Cancer; Drug Delivery 209 Synthetic Gene Delivery Systems 590 Tandem Bispecific Chimeric Antigen Receptors 392 Targeted Gene Expression 723 Tumor-Specific Promoter 677 U1 Adaptors, iRGD, KRAS, MYC, Pancreatic Cancer 262 Vector Characterization/Biology 655 Vector Immunology/Host Responses 208, 415, 517 Cancer - T-Cell Therapy AAV Vectors Adenovirus Cytokine Adenovirus Vectors
323 405 410, 513 S325
KEYWORD INDEX Animal Models 275 Anti CD19 CAR T KTE-C19 745 Cancer - Cell Therapy 192, 199, 204, 393, 402, 515, 646, 652, 745, 748 Cancer - Gene Therapy 74, 192, 206, 323, 405, 506, 653, 746, 747, 752, 762 Cancer - Targeted Therapy 202, 204, 392, 393, 396, 505, 762 CAR T Cells; Lymphoma; Manufacturing 287 CD19, Sleeping Beauty 278 Cell Delivery 596 Cell Processing 287, 402, 455, 650, 714 Cell Therapy - ES Cells and iPS Cells 236 Cell Therapy - Hematopoietic 236 Cell Therapy - Mesenchymal 466 Checkpoint Inhibitor 513 Chimeric Antigen Receptor T Cells 204 Chimeric Antigen Receptors 69, 73, 74, 190, 193, 195, 198, 200, 203, 210, 247, 277, 392, 394, 397, 398, 400, 406, 451, 507, 510, 512, 515, 640, 649, 650, 651, 676, 745, 747, 748, 751 Clinical Gene Therapy 247, 272 Clonality, T-Cell Repertoire 232 CRISPR/Cas9 404, 572 DNA Viral Vectors 194 Gene Correction/Modification/Targeting 121, 391, 394, 398, 572 Genetic Diseases 232 Glycobiology 510 Good Manufacturing Procedures 75, 203, 466, 714 Hematologic Diseases 71, 213, 400, 637 IL13Ra2, GBM, Solid Tumors 76 IL-4 751 Immunotherapy 71, 73, 75, 76, 190, 191, 193, 194, 195, 197, 198, 199, 202, 203, 206, 232, 272, 275, 277, 278, 287, 391, 405, 406, 410, 451, 455, 505, 507, 510, 512, 513, 637, 638, 640, 646, 648, 649, 651, 652, 653, 746, 751 In Vivo T Cell Engineering 506 Inflammatory Toxicity 197 Lentiviral Vectors 195 Lentivirus Vectors 121, 404, 638, 648 Leukemia and Lymphoma 69, 210 Meganuclease, AAV vectors 394 Meganucleases 200 Multiple Myeloma 752 Mutated Neoantigens 391 Nanobiotechnology 396 Non-Viral Gene Delivery 278 Non-Viral Vectorology 213 Oncolytic Adenovirus 515 Oncolytic Virus 68 PD-1 572 Pediatric Cancers - Solid Tumors 649 Process Modeling; Monte Carlo Simulations 650 RNA Viral Vectors 68, 506 Sleeping Beauty Transposition; Minicircle 73 TALENs 397, 676 Tandem Bispecific Chimeric Antigen Receptors 392 Targeted Gene Expression 76 Vaccines 68, 191, 596 WT1 193 Zinc-Finger Nucleases 752 S326
Cancer Stem Cells, Tumor Microenvironment Cancer - Cell Therapy Gene Expression Targeted Gene Expression
679 679 679
Capsid Engineering AAV Vectors Gene Expression Neurological Disorders
252 252 252
Capsid Modification AAV Vectors Vector Production
95 95
CAR T Cells; Lymphoma; Manufacturing Cancer - T-Cell Therapy 287 Cell Processing 287 Immunotherapy 287 Cardiopulmonary Animal Models Clinical Gene Therapy Genetic Diseases
383 383 383
Cardiovascular Diseases AAV Vectors 89, 180, 297 Adenovirus Vectors 179 Animal Models 180, 447, 448 Cancer - Targeted Therapy 89 Cell Delivery 174, 178 Cell Therapy - Endothelial 174 Cell Therapy - ES Cells and iPS Cells 244 Cell Therapy - Mesenchymal 246, 448 Cell Therapy - Other 174, 178, 447 Chemical Conjugates 177 Clinical Gene Therapy 297 CRISPR/Cas9 20 Familial Hypercholesterolaemia 19 Gene Expression 172, 177 Genetic Diseases 20, 358 Long Term Expression 172 Metabolic Diseases 179, 358 MicroRNA 19 MitoTimer 358 Non-Viral Gene Delivery 19 Notch Signaling 447 QC/QA Assays 244 Tissue Engineering 246 CCR5 AAV Vectors 43 CRISPR/Cas9 43 Gene Correction/Modification/Targeting 43 CD19, Sleeping Beauty Cancer - T-Cell Therapy 278 Immunotherapy 278 Non-Viral Gene Delivery 278 CD40L, X-HIGM, Gene Editing AAV Vectors 316 Hematopoietic Stem Cells 316 TALENs 316 Molecular Therapy Volume 24, Supplement 1, May 2016 Copyright © The American Society of Gene & Cell Therapy
KEYWORD INDEX CD8+ T Cells, Ovalbumin AAV Vectors Infectious Diseases Vector Immunology/Host Responses
79 79 79
Cell Delivery AAV Vectors 554 Adenovirus Vectors 420 Cancer - Cell Therapy 242, 420, 442, 511 Cancer - T-Cell Therapy 596 Cardiovascular Diseases 174, 178 Cell Therapy - Endothelial 174 Cell Therapy - Hematopoietic 336, 521, 603 Cell Therapy - Lung 21 Cell Therapy - Mesenchymal 242, 554 Cell Therapy - Neuronal 521 Cell Therapy - Other 174, 178, 340 CRISPR/Cas9 496, 603 Duchenne Muscular Dystrophy; Exon Skipping 584 Exosomes 496 Genetic Diseases 21, 336 Immunotherapy 511 Nanobiotechnology 442 Neuromuscular Disorders 584 Oligonucleotide Based Therapies 584 Tissue Engineering 340 Tunneling Nanotubes 336 Vaccines 596 Cell Processing Bioprocess 343 Cancer - Cell Therapy 402, 459 Cancer - T-Cell Therapy 287, 402, 455, 650, 714 CAR T Cells; Lymphoma; Manufacturing 287 Cell Reprogramming 343 Cell Therapy - Hematopoietic 526 Cell Therapy - Mesenchymal 245 Cell Therapy - Neuronal 375 Chimeric Antigen Receptors 650 Genetic Diseases 425 Good Manufacturing Procedures 459, 714 GPCR, TDP-43, Cannabinoids 375 Hematopoietic Stem Cells 526 Immunotherapy 287, 425, 455 Lentivirus Vectors 707 MicroRNA 245 Non-Viral Gene Delivery 343 Pharmacology/Toxicology 375 Process Modeling; Monte Carlo Simulations 650 T-Cell Development, Infections, Notch, Thymus 425 Vector Production 707 Cell Reprogramming AAV Vectors 266 Bioprocess 343 Cancer - Cell Therapy 645 Cell Processing 343 Cell Therapy - Muscle 525 Cell Therapy - Other 645 CRISPR/Cas9 266 Gen Expression 704 Gene Correction/Modification/Targeting 40 Molecular Therapy Volume 24, Supplement 1, May 2016 Copyright © The American Society of Gene & Cell Therapy
Lentivirus Vectors Musculo-Skeletal Disorders Non-Viral Gene Delivery Targeted Gene Expression Vector Characterization/Biology Vector Production Cell Therapy - Endothelial Cardiovascular Diseases Cell Delivery Cell Therapy - Other
704 525 343, 344 40 344 704 174 174 174
Cell Therapy - ES Cells and iPS Cells Cancer - Cell Therapy 528 Cancer - T-Cell Therapy 236 Cardiovascular Diseases 244 Cell Therapy - Hematopoietic 234, 236 Cell Therapy - Muscle 565, 628 Clinical Gene Therapy 527 CRISPR/Cas9 527, 730 Gene Correction/Modification/Targeting 122, 243, 312, 332, 347, 730 Genetic Diseases 522 Hematologic Diseases 444, 522 Induced Pluripotent Stem Cells 241, 243, 444 Lentivirus Vector 234 Lentivirus Vectors 241 Lentivirus, DMD 565 Long Term Expression 234 Metabolic Diseases 347 Musculo-Skeletal Disorders 312 Neurological Disorders 528 Neuromuscular Disorders 730 Non-Viral Gene Delivery 628 QC/QA Assays 244 Respiratory/Lung Disorders 122 TALENs 332, 565 Transposon 628 Urea Cycle Disorder 347 Cell Therapy - Hematopoietic AAV Vectors 123, 285 Adenovirus Vectors 757 Animal Models 427 Cancer - Cell Therapy 233, 273 Cancer - Gene Therapy 523 Cancer - T-Cell Therapy 236 Cell Delivery 336, 521, 603 Cell Processing 526 Cell Therapy - ES Cells and iPS Cells 234, 236 Cell Therapy - Neuronal 521 Cell Therapy - Other 763 Clinical Gene Therapy 235 CRISPR/Cas9 603 Ex-Vivo Expansion 523 Foamy Virus 683 Gene Correction/Modification/Targeting 123, 432, 562 Gene Expression 766 Genetic Diseases 163, 336 Genetic Modification 734 Hematologic Diseases 233, 683, 763 S327
KEYWORD INDEX Hematopoietic Stem Cells 235, 273, 283, 285, 432, 523, 526, 562, 689, 734, 757 HIV 427, 763 Immunotherapy 239, 427, 685 Infectious Diseases 685 Lentivirus Vector 234 Lentivirus Vectors 283, 766 Long Term Expression 234, 689 Luekocyte Adhesion Deficiency 285 Macrophages, HSC 766 Metabolic Diseases 163 Neurological Disorders 374 RNA Viral Vectors 6, 374, 683 TALEN 562 Tissue Engineering 239 Tunneling Nanotubes 336 Vector Production 6 Zinc-Finger Nucleases 734 Cell Therapy - Lung Animal Models Cancer - Gene Therapy Cell Delivery Cell Therapy - Other Genetic Diseases Non-Small Cell Lung Cancer Pulmonary Macrophage Transplantation Respiratory/Lung Disorders RNA Viral Vectors RNAi and shRNA Vector Immunology/Host Responses
182 585 21 35 21 585 35 35, 182 438 585 438
Cell Therapy - Mesenchymal AAV Vectors 524, 554, 632 Adenovirus Vectors 453 Animal Models 448 Cancer - Cell Therapy 242 Cancer - T-Cell Therapy 466 Cardiovascular Diseases 246, 448 Cell Delivery 242, 554 Cell Processing 245 Cell Therapy - Muscle 238 Clinical Gene Therapy 453 Diabetes 354 Gene Correction/Modification/Targeting 354 Good Manufacturing Procedures 466 Lentivirus Vectors 632 Metabolic Diseases 354 MicroRNA 245 Musculo-Skeletal Disorders 238 Osteoporosis, Alpha-1 Antitrypsin 632 Tissue Engineering 246, 524 Cell Therapy - Muscle AAV Vectors 85 Animal Models 635 Cancer - Cell Therapy 636 Cell Reprogramming 525 Cell Therapy - ES Cells and iPS Cells 565, 628 Cell Therapy - Mesenchymal 238 Cell Therapy - Other 446 CRISPR/Cas9 577 S328
Gene Expression 449 Lentivirus Vectors 240 Lentivirus, DMD 565 Musculo-Skeletal Disorders 238, 446, 449, 525, 577, 635, 636 Neuromuscular Disorders 85 Non-Viral Gene Delivery 628 TALENs 565 Tissue Engineering 240 Transposon 628 Cell Therapy - Neuronal AAV Vectors 99, 678 Cancer - Cell Therapy 678 Cancer - Targeted Therapy 208, 443 Cell Delivery 521 Cell Processing 375 Cell Therapy - Hematopoietic 521 Gene Expression 99 GPCR, TDP-43, Cannabinoids 375 Neural Stem Cells, Pediatric Brain Tumors 443 Pharmacology/Toxicology 375 Promoters 99 Vector Immunology/Host Responses 208 Cell Therapy - Other Animal Models 430, 447 Cancer - Cell Therapy 645 Cardiovascular Diseases 174, 178, 447 Cell Delivery 174, 178, 340 Cell reprogramming 645 Cell Therapy - Endothelial 174 Cell Therapy - Hematopoietic 763 Cell Therapy - Lung 35 Cell Therapy - Muscle 446 Commercialisation 452 Commercialization 718 CRISPR/Cas9 573 Gene Correction/Modification/Targeting 573 Gene Expression 454 Genetic Diseases 161 Good Manufacturing Procedures 445 Hematologic Diseases 763 HIV 763 Immunotherapy 426 Infectious Diseases 426 Long Term Expression 161 Lysosomal Storage Deseases, Enzyme Replacement 430 Magnetic Targeting, MRI 338 Metabolic Diseases 430 MicroRNA 338 Musculo-Skeletal Disorders 446, 454 Non-Viral Gene Delivery 338 Notch Signaling 447 Ophthalmic Diseases 445 Pulmonary Macrophage Transplantation 35 Regulatory Issues 718 Respiratory/Lung Disorders 35 Tissue Engineering 340, 452 Cellular Clearance Gene Expression Ophthalmic Diseases
187 187 Molecular Therapy Volume 24, Supplement 1, May 2016 Copyright © The American Society of Gene & Cell Therapy
KEYWORD INDEX CGD Stem Cell Gene Correction CRISPR/Cas9 41 Gene Correction/Modification/Targeting 41 Non-Viral Gene Delivery 41 Checkpoint Inhibitor Adenovirus Vectors 513 Cancer - T-Cell Therapy 513 Immunotherapy 513 Chemical Conjugates Apoptosis 345 Cancer - Gene Therapy 345, 602 Cardiovascular Diseases 177 Curcumin, CDC, Breast Cancer 345 Gene Expression 177 Immunotherapy 225 Lentivirus Vectors 225 Nanobiotechnology 342 Ophthalmic Diseases 342 Retrovirus Vectors 602 T-Cells 225 Chimeric Antigen Receptor T Cells Cancer - Cell Therapy Cancer - Targeted Therapy Cancer - T-Cell Therapy
204 204 204
Chimeric Antigen Receptor, CAR Cancer - Gene Therapy Cancer - Targeted Therapy
670 670
Chimeric Antigen Receptors AAV Vectors 251 Animal Models 750 Anti CD19 CAR T KTE-C19 745 Cancer - Cell Therapy 207, 515, 745, 748 Cancer - Gene Therapy 74, 207, 747 Cancer - Targeted Therapy 392 Cancer - T-Cell Therapy 69, 73, 74, 190, 193, 195, 198, 200, 203, 210, 247, 277, 392, 394, 397, 398, 400, 406, 451, 507, 510, 512, 515, 640, 649, 650, 651, 676, 745, 747, 748, 751 Cell Processing 650 Clinical Gene Therapy 247 CRISPR/Cas9 274 Gene Correction/Modification/Targeting 394, 398, 761 Glycobiology 510 Good Manufacturing Procedures 203 Hematologic Diseases 400, 407 Hemophilia 434 IL-4 751 Immunotherapy 73, 190, 193, 195, 198, 203, 274, 277, 399, 406, 434, 451, 507, 510, 512, 640, 649, 651, 750, 751, 761 Lentiviral Vectors 195 Lentivirus Vectors 407 Leukemia and Lymphoma 69, 210 Meganuclease, AAV Vectors 394 Meganucleases 200 Non-Viral Gene Delivery 705 Oncolytic Adenovirus 515 Pediatric Cancers - Solid Tumors 649 Molecular Therapy Volume 24, Supplement 1, May 2016 Copyright © The American Society of Gene & Cell Therapy
Process Modeling; Monte Carlo Simulations 650 Regulatory T Cell 434 Sleeping Beauty Transposition; Minicircle 73 TALENs 397, 676 Tandem Bispecific Chimeric Antigen Receptors 392 Targeting 251 Toxicology Studies 399 Transposon 705 WT1 193 Chromatography AAV Vectors Good Manufacturing Procedures Vector Production
100 100 100
Chronic Pain HSV Vectors Neurological Disorders Targeted Gene Expression
111 111 111
Clinical Gene Therapy AAV Vectors 24, 83, 185, 248, 249, 297, 387, 470, 480, 497, 547, 592, 615, 697, 715, 755 Adenovirus Vectors 36, 109, 110, 112, 115, 116, 453, 514, 661 Adrenoleukodystrophy 250 Animal Models 383, 615 Assay Development 171 Bioinformatics 537 Cancer - Gene Therapy 61, 514 Cancer - Targeted Therapy 72, 661 Cancer - T-Cell Therapy 247, 272 Cardiopulmonary 383 Cardiovascular Diseases 297 Cell Therapy - ES Cells and iPS Cells 527 Cell Therapy - Hematopoietic 235 Cell Therapy - Mesenchymal 453 Chimeric Antigen Receptors 247 CRISPR/Cas9 326, 527, 731 Cystic Fibrosis 711 Gene Correction/Modification/Targeting 280, 326, 731 Gene Expression 109, 110, 112, 115, 116, 171 Genetic Diseases 83, 224, 353, 383, 534, 755 Good Manufacturing Procedures 592 HCV, AAV 258 Hematologic Diseases 279, 280 Hematopoietic Stem Cells 235, 314, 364, 754 Hemophilia 171 High Quality Grade 592 Human Immune Response to AAV 24 Immunotherapy 61, 272 Infectious Diseases 258 Integration Sites 531 Keloid 112 Lentivirus Vectors 250, 279, 364, 531, 534, 711, 754 Long Term Expression 24 Mathematical Modeling 314 Mesothelioma 661 Metabolic Diseases 353 MicroRNA 282 Musculo-Skeletal Disorders 328, 387, 480, 497 Natural History and Trial Development 715 Neurological Disorders 250, 267, 367 S329
KEYWORD INDEX Neuromuscular Disorders 715 Non-Viral Gene Delivery 72 Oligonucleotide Based Therapies 328 Ophthalmic Diseases 83, 248, 249, 267 Precision Medicine 537 Regulatory Issues 719 Retroviral Replicating Vector 61 Retrovirus Vectors 224, 367, 531 RNA Viral Vectors 537 RNAi and shRNA 258 Spinal Muscular Atrophy, Type 1 480 Sporadic Inclusion Body Myositis, Follistatin 497 Targeted Gene Expression 282 Toxicology Studies 470 Vaccines 36 Vector Characterization/Biology 547 Vector Immunology/Host Responses 185, 697 Vector Production 711 Wet Age-Related Macular Degeneration 248 Clonality, T-Cell Repertoire Cancer - T-Cell Therapy 232 Genetic Diseases 232 Immunotherapy 232 Colorectal Cancer Animal Models Cancer - Gene Therapy DNA Viral Vectors Gene Expression HSV Vectors Targeted Gene Expression
675 664 664 675 664 675
Commercialisation Cell Therapy - Other Tissue Engineering
452 452
Commercialization Cell Therapy - Other Regulatory Issues
718 718
Common Rule Ethical Issues Regulatory Issues
717 717
Continuous Flow Ultracentrifugation AAV Vectors DNA Viral Vectors Vector Production
97 97 97
CRISPR/Cas AAV Vectors 569 Gene Correction/Modification/Targeting 569 TALENs 569 CRISPR/Cas9 AAV Vectors 39, 43, 119, 120, 266, 293, 303, 321, 322, 481, 482, 543, 570 Adenovirus Vectors 15, 104 Alzheimer’s Disease 567 Animal Models 167 Cancer - Cell Therapy 422 Cancer - T-Cell Therapy 404, 572 Cardiovascular Diseases 20 S330
CCR5 43 Cell Delivery 496, 603 Cell Reprogramming 266 Cell Therapy - ES Cells and iPS Cells 527, 730 Cell Therapy - Hematopoietic 603 Cell Therapy - Muscle 577 Cell Therapy - Other 573 CGD Stem Cell Gene Correction 41 Chimeric Antigen Receptors 274 Clinical Gene Therapy 326, 527, 731 Diabetes 543 Dystrophin 561 Epigenetic Regulation 131, 385, 729 Exosomes 313, 496 Gene Correction/Modification/Targeting 3, 15, 39, 41, 43, 119, 120, 124, 126, 128, 131, 134, 136, 137, 139, 140, 303, 313, 317, 326, 329, 330, 334, 481, 482, 487, 488, 501, 504, 543, 559, 561, 567, 568, 570, 571, 572, 573, 575, 576, 729, 730, 731, 732, 733 Gene Expression 334 Genetic Diseases 20, 481, 488, 561, 568, 576, 733 Genomic Deletions 135 Hematologic Diseases 140, 559, 571, 692 Hematopoietic Stem Cells 135, 330, 564, 732, 768 Hemophilia 128 HIV Latency 125 HIV-1, Virual Escape 331 Homology-Directed Repair 568 HSC Engraftment, Hematologic Disease 732 Immunotherapy 274, 422, 768 Induced Pluripotent Stem Cells 318, 557, 626, 735 Infectious Diseases 125, 331, 574, 768 Lentiviral Vectors 322 Lentivirus Vectors 3, 126, 134, 135, 327, 331, 404, 532, 564, 574 Liver 120 Musculo-Skeletal Disorders 104, 139, 317, 322, 385, 501, 504, 577, 735 Neurological Disorders 321, 327, 567 Neuromuscular Disorders 730 Non-Viral Gene Delivery 41, 335, 487 Ophthalmic Diseases 124, 329 PD-1 572 Precision Medicine 141, 575 QC/QA Assays 141 Respiratory/Lung Disorders 23, 167 RNA Delivery 335 RNAi and shRNA 23, 532 Synthetic Biology 136 TALENs 136, 137, 139, 692 Targeted Epigenetic Editing 729 Targeted Gene Expression 125, 557, 626 Thalassemia 692 Tissue Engineering 318 Vector Characterization/Biology 293 Crude Lysate AAV Vectors QC/QA Assays Vector Production
290 290 290
Molecular Therapy Volume 24, Supplement 1, May 2016 Copyright © The American Society of Gene & Cell Therapy
KEYWORD INDEX Curcumin, CDC, Breast Cancer Apoptosis 345 Cancer - Gene Therapy 345 Chemical Conjugates 345 Cystic Fibrosis Clinical Gene Therapy Lentivirus Vectors Vector Production
711 711 711
Cytokines Animal Models Gene Expression
441 441
Cytotoxic Transgene AAV Vectors Cancer - Gene Therapy
215 215
Database Development AAV Vectors Good Manufacturing Procedures Vector Production
591 591 591
Delivery Techniques AAV Vectors Ophthalmic Diseases Targeted Gene Expression Design of Experiment Lentivirus Vectors Vector Production
29 29 29 708 708
Diabetes AAV Vectors 50, 543 Animal Models 50 Cell Therapy - Mesenchymal 354 CRISPR/Cas9 543 Gene Correction/Modification/Targeting 354, 543 Long Term Expression 50 Metabolic Diseases 354 DMD Dogs AAV Vectors Animal Models Neuromuscular Disorders
721 721 721
DNA Vaccines, Electroporation Cancer - Targeted Therapy 401 Immunotherapy 401 DNA Vaccines Non-Viral Gene Delivery 436 Vaccines 436 DNA Viral Vectors AAV Vectors 87, 97, 102, 253, 254, 294, 306, 311, 319, 549, 630, 727 Adenovirus Vectors 13, 14, 17, 102, 106 Cancer - Gene Therapy 419, 520, 664 Cancer - Targeted Therapy 13, 419 Cancer - T-Cell Therapy 194 Colorectal Cancer 664 Continuous Flow Ultracentrifugation 97 Gene Correction/Modification/Targeting 311, 319 Molecular Therapy Volume 24, Supplement 1, May 2016 Copyright © The American Society of Gene & Cell Therapy
Hematopoietic Stem Cells 106 Hemophilia 253 HSV Vectors 107, 520, 664 Immunotherapy 194 Minicircle 306 Musculo-Skeletal Disorders 630 Oncolytic Vectors 520 Ophthalmic Diseases 306 Targeted Gene Expression 108 Vaccines 712 Vector Characterization/Biology 14, 17, 107, 108, 254, 727 Vector Production 87, 97, 294, 549, 712 Vector Purification 294 Down Syndrome Genetic Diseases Neurological Disorders RNAi and shRNA
53 53 53
Duchenne Muscular Dystrophy; Exon Skipping Cell Delivery Neuromuscular Disorders Oligonucleotide Based Therapies
584 584 584
Dystrophin CRISPR/Cas9 561 Gene Correction/Modification/Targeting 561 Genetic Diseases 561 Enteric Nervous System, Gastrointestinal Disorders AAV Vectors Animal Models Neurological Disorders Epigenetic Regulation AAV Vectors CRISPR/Cas9 Gene Correction/Modification/Targeting Musculo-Skeletal Disorders Targeted Epigenetic Editing Vector Characterization/Biology Esophageal Squamous Cell Carcinoma Animal Models Cancer - Gene Therapy Cancer - Targeted Therapy
612 612 612
8 131, 385, 729 131, 729 385 729 8 412 412 412
Ethical Issues Animal Models 627 Common Rule 717 Ethical Issues 717 Gene Correction/Modification/Targeting 720 Nanobiotechnology 720 Neurological Disorders 627 Regulatory Issues 717 Evolution and Development AAV Vectors Neuromuscular Disorders Vector Characterization/Biology
389 389 389
Exon Skipping AAV Vectors Musculo-Skeletal Disorders
624 624 S331
KEYWORD INDEX Exosomes AAV Vectors Cell Delivery CRISPR/Cas9 Gene Correction/Modification/Targeting Non-Viral Gene Delivery Ophthalmic Diseases RNA Delivery Vector Characterization/Biology Vector Immunology/Host Responses Vector Transduction
595 496 313, 496 313 341 594 341 594 595 594
Ex-Vivo Expansion Cancer - Gene Therapy Cell Therapy - Hematopoietic Hematopoietic Stem Cells
523 523 523
Familial Hypercholesterolaemia Cardiovascular Diseases MicroRNA Non-Viral Gene Delivery
19 19 19
Fanconi Anemia Gene Correction/Modification/Targeting Genetic Diseases Hematopoietic Stem Cells Foamy Virus Cell Therapy - Hematopoietic Hematologic Diseases Hematopoietic Stem Cells RNA Viral Vectors Vector Characterization/Biology
753 753 753 683 683 1 1, 683 1
FSHD Musculo-Skeletal Disorders Oligonucleotide Based Therapies Targeted Gene Expression
153 153 153
Functional MRI (fMRI), Visual Pathways, Brain AAV Vectors Genetic Diseases Ophthalmic Diseases
265 265 265
Gastrointestinal Inflammation Animal Models Gene Expression Metabolic Diseases
477 477 477
Gen Expression Cell Reprogramming Lentivirus Vectors Vector Production
704 704 704
Gene Correction/Modification/Targeting AAV Vectors 39, 43, 44, 48, 86, 93, 119, 120, 123, 129, 133, 165, 188, 295, 303, 311, 319, 359, 481, 482, 483, 485, 542, 543, 569, 570, 605, 620, 725 Adenovirus Vectors 15, 18 Alzheimer’s Disease 567 Animal Models 38, 284, 725 Barcoding 542 Cancer - Cell Therapy 641 Cancer - T-Cell Therapy 121, 391, 394, 398, 572 S332
CCR5 43 Cell Reprogramming 40 Cell Therapy - ES Cells and iPS Cells 122, 243, 312, 332, 347, 730 Cell Therapy - Hematopoietic 123, 432, 562 Cell Therapy - Mesenchymal 354 Cell Therapy - Other 573 CGD Stem Cell Gene Correction 41 Chimeric Antigen Receptors 394, 398, 761 Clinical Gene Therapy 280, 326, 731 CRISPR/Cas 569 CRISPR/Cas9 3, 15, 39, 41, 43, 119, 120, 124, 126, 128, 131, 134, 136, 137, 139, 140, 303, 313, 317, 326, 329, 330, 334, 481, 482, 487, 488, 501, 504, 543, 559, 561, 567, 568, 570, 571, 572, 573, 575, 576, 729, 730, 731, 732, 733 Diabetes 354, 543 DNA Viral Vectors 311, 319 Dystrophin 561 Epigenetic Regulation 131, 729 Ethical Issues 720 Exosomes 313 Fanconi Anemia 753 Gene Expression 334, 560 Genetic Diseases 165, 226, 325, 481, 488, 558, 561, 568, 576, 579, 601, 733, 753 Glaucoma 188 Hematologic Diseases 42, 118, 140, 280, 559, 560, 571, 682 Hematopoietic Stem Cells 37, 42, 330, 432, 558, 562, 732, 753 Hemophilia 128 HIV 44 Homology-Directed Repair 568 HSC Engraftment, Hematologic Disease 732 Immunodeficiency 325 Immunotherapy 132, 391, 761 Induced Pluripotent Stem Cells 243 Infectious Diseases 18, 44, 765 Inflammation 226 Lentivirus Vectors 3, 121, 126, 127, 129, 130, 134, 226, 286, 533, 538, 566, 682, 764 Liver 120 Meganuclease, AAV Vectors 394 Meganucleases 138, 579 Metabolic Diseases 48, 86, 93, 347, 354, 359 MicroRNA 742 MPS1 93 Mucopolysaccharidosis 485 Musculo-Skeletal Disorders 139, 312, 317, 483, 501, 504 Mutated Neoantigens 391 Nanobiotechnology 563, 720 Neurological Disorders 295, 567, 605, 742 Neuromuscular Disorders 138, 730 Non-Viral Gene Delivery 41, 487, 601 Non-Viral Vectorology 533 Ophthalmic Diseases 124, 188, 329, 620 PD-1 572 Precision Medicine 575 Promoterless Vector 48 Respiratory/Lung Disorders 122 Retrovirus Vectors 284 RNA Delivery 538 Molecular Therapy Volume 24, Supplement 1, May 2016 Copyright © The American Society of Gene & Cell Therapy
KEYWORD INDEX Synthetic Biology 136 TALEN 562 TALENs 132, 136, 137, 139, 325, 332, 569 Targeted Epigenetic Editing 729 Targeted Gene Expression 40, 315, 542 Urea Cycle Disorder 347 Vector Immunology/Host Responses 764 Vector Production 286 XIST-Mediated Chromosome Silencing 315 Zinc-Finger Nucleases 37, 38, 118, 127, 130, 133, 315, 485, 563, 566, 641, 765 Gene Expression AAV 499 AAV Vectors 10, 11, 60, 91, 98, 99, 158, 159, 164, 252, 270, 292, 299, 310, 370, 456, 471, 474, 545, 546, 608, 610, 616, 618, 622, 700, 701, 726, 728 Adenovirus Vectors 109, 110, 112, 113, 115, 116, 324, 413 Animal Models 64, 441, 477, 499, 675, 749 Assay Development 171 brain Delivery 618 Butyrylcholinesterase 60 Cancer - Cell Therapy 216, 679 Cancer - Gene Therapy 113, 142, 413 Cancer - Targeted Therapy 216 Cancer Stem Cells, Tumor Microenvironment 679 Capsid Engineering 252 Cardiovascular Diseases 172, 177 Cell Therapy - Hematopoietic 766 Cell Therapy - Muscle 449 Cell Therapy - Neuronal 99 Cell Therapy - Other 454 Cellular Clearance 187 Chemical Conjugates 177 Clinical Gene Therapy 109, 110, 112, 115, 116, 171 Colorectal Cancer 675 CRISPR/Cas9 334 Cytokines 441 Gastrointestinal Inflammation 477 Gene Correction/Modification/Targeting 334, 560 Genetic Diseases 164, 373 Hematologic Diseases 560 Hemophilia 5, 171, 456, 545 Immunotherapy 64, 142, 749 Infectious Diseases 681 Inflammation 146 Insertional Mutagenesis 681 Keloid 112 Lentivirus Vectors 5, 609, 728, 766 Long Term Expression 60, 172, 324, 609 Macrophages, HSC 766 Metabolic Diseases 158, 159, 477 MicroRNA 146 Minicircle 743 Muscular Dystrophy 726 Musculo-Skeletal Disorders 449, 454, 499, 622, 726 Neurological Disorders 252, 370, 373, 610, 616, 618, 736 Neurotrophic Factor, Parkinson 610 Non-Viral Vectorology 289, 743 Oligonucleotide Based Therapies 146 Ophthalmic Diseases 98, 187, 270, 320, 608, 736 Optogenetic Therapy 270 Molecular Therapy Volume 24, Supplement 1, May 2016 Copyright © The American Society of Gene & Cell Therapy
Pharmacology/Toxicology 299 Promoters 99 Reactive Oxygen Species 216 Regulation 546 Respiratory/Lung Disorders 743 RNA Viral Vectors 681 Targeted Gene Expression 320, 675, 679 Transgene Engineering 456 Vector Characterization/Biology 10, 11, 91, 289, 474, 546 Vector Immunology/Host Responses 310, 700, 701 Vector Production 292, 471 Gene Therapy AAV Vectors HSV Vectors Vector Production
94 94 94
Genetic Diseases AAV 25 AAV Vectors 27, 28, 45, 49, 57, 83, 101, 164, 165, 257, 265, 269, 300, 348, 350, 381, 481, 551, 606, 617, 621, 755 Adenovirus Vectors 47 Amyotrophic Lateral Sclerosis 606 Animal Models 166, 183, 350, 357, 383, 631 Auditory Diseases 269 Bone Defect 631 Cancer - T-Cell Therapy 232 Cardiopulmonary 383 Cardiovascular Diseases 20, 358 Cell Delivery 21, 336 Cell Processing 425 Cell Therapy - ES Cells and iPS Cells 522 Cell Therapy - Hematopoietic 163, 336 Cell Therapy - Lung 21 Cell Therapy - Other 161 Clinical Gene Therapy 83, 224, 353, 383, 534, 755 Clonality, T-Cell Repertoire 232 CRISPR/Cas9 20, 481, 488, 561, 568, 576, 733 Down Syndrome 53 Dystrophin 561 Fanconi Anemia 753 Functional MRI (fMRI), Visual Pathways, Brain 265 Gene Correction/Modification/Targeting 165, 226, 325, 481, 488, 558, 561, 568, 576, 579, 601, 733, 753 Gene Expression 164, 373 GM2 Gangliosidosis 363 Hearing 269 Hematologic Diseases 522, 581 Hematopoietic Stem Cells 32, 291, 558, 753 Homology-Directed Repair 568 Immunodeficiency 325 Immunotherapy 232, 425 Inflammation 226 Krabbe Disease 57 Lentivirus Vectors 226, 291, 534, 631 Liver Diseases 183 Long Term Expression 161, 439 Lysosomal Storage Disease 348 Meganucleases 579 Metabolic Diseases 25, 46, 47, 160, 163, 166, 169, 353, 357, 358, 360, 369 MicroRNA 606 S333
KEYWORD INDEX MitoTimer 358 Musculo-Skeletal Disorders 49, 381, 741 Neurological Disorders 25, 27, 45, 53, 57, 160, 348, 360, 363, 369, 373, 621, 741 Neuromuscular Disorders 150, 551 Non-Viral Gene Delivery 169, 439, 597, 601 Oligonucleotide Based Therapies 150, 581, 741 Ophthalmic Diseases 28, 46, 83, 265, 617 Pompe Disease 49, 381 Respiratory/Lung Disorders 183, 597 Retrovirus Vectors 224 RNAi and shRNA 53 Routes of Administration 300 Splice, X-Linked Agammaglobulinemia 581 TALENs 325 Targeted Gene Expression 300 T-Cell Development, Infections, Notch, Thymus 425 Toxicology Studies 32 Tunneling Nanotubes 336 Urea Cycle Disorder 45 Vector Characterization/Biology 257 Vector Production 101 XLRP 101 Genetic Modification Cell Therapy - Hematopoietic Hematopoietic Stem Cells Zinc-Finger Nucleases
734 734 734
Genomic Deletions CRISPR/Cas9 135 Hematopoietic Stem Cells 135 Lentivirus Vectors 135 Glaucoma AAV Vectors Gene Correction/Modification/Targeting Ophthalmic Diseases
188 188 188
Glioma Cancer - Targeted Therapy HSV Vectors Neurological Disorders
665 665 665
Glycobiology Cancer - T-Cell Therapy 510 Chimeric Antigen Receptors 510 Immunotherapy 510 GM2 Gangliosidosis Genetic Diseases Neurological Disorders GMP Manufacturing AAV Vectors Good Manufacturing Procedures Vector Production
363 363 460, 710 460, 710 460, 710
Good Manufacturing Procedures AAV Vectors 100, 308, 309, 460, 463, 591, 592, 710 Adenovirus Vectors 706, 713 Cancer - Cell Therapy 459 Cancer - T-Cell Therapy 75, 203, 466, 714 Cell Processing 459, 714 S334
Cell Therapy - Mesenchymal 466 Cell Therapy - Other 445 Chimeric Antigen Receptors 203 Chromatography 100 Clinical Gene Therapy 592 Database Development 591 GMP Manufacturing 460, 710 High Quality Grade 592 Immunotherapy 75, 203 Ophthalmic Diseases 445 Orbital Shaken Bioreactors 463 Vector Production 100, 308, 309, 460, 463, 591, 706, 710, 713 GPCR, TDP-43, Cannabinoids Cell Processing 375 Cell Therapy - Neuronal 375 Pharmacology/Toxicology 375 HBV, AAV Infectious Diseases Long Term Expression RNAi and shRNA
260 260 260
HCV, AAV Clinical Gene Therapy Infectious Diseases MicroRNA Regulatory Issues RNAi and shRNA
258 258 143 143 143, 258
Hearing AAV Vectors Auditory Diseases Genetic Diseases
269 269 269
Hematologic Diseases AAV Vectors Animal Models Cancer - Cell Therapy Cancer - Gene Therapy Cancer - Targeted Therapy Cancer - T-Cell Therapy Cell Therapy - ES Cells and iPS Cells Cell Therapy - Hematopoietic Cell Therapy - Other Chimeric Antigen Receptors Clinical Gene Therapy CRISPR/Cas9 Foamy Virus Gene Correction/Modification/Targeting
227, 760 227, 760 233, 276 677 65, 663, 677 71, 213, 400, 637 444, 522 233, 683, 763 763 400, 407 279, 280 140, 559, 571, 692 683 42, 118, 140, 280, 559, 560, 571, 682 Gene Expression 560 Genetic Diseases 522, 581 Hematopoietic Stem Cells 4, 42, 221, 228, 229, 231, 281, 508, 759 Hemophilia 227, 760 HIV 763 Immunotherapy 71, 276, 508, 637, 663 Induced Pluripotent Stem Cells 444 Lentivirus Vectors 4, 221, 228, 229, 230, 279, 281, 407, 682, 686, 690, 759 Mitochondrial Biogenesis 65 Mixed Chimerism 231 Molecular Therapy Volume 24, Supplement 1, May 2016 Copyright © The American Society of Gene & Cell Therapy
KEYWORD INDEX Non-Viral Vectorology 213 Oligonucleotide Based Therapies 581 Pharmacology/Toxicology 690 QC/QA Assays 473 RNA Viral Vectors 683 Splice, X-Linked Agammaglobulinemia 581 TALENs 686, 692 Telomerase 276 Thalassemia 692 Tumor-Specific Promoter 677 Vector Characterization/Biology 230, 473 Zinc-Finger Nucleases 118 Hematopoietic Stem Cells AAV Vectors 285, 316 Adenovirus Vectors 106, 757 Animal Models 758, 767 Antibody Delivery 429 Cancer - Cell Therapy 273 Cancer - Gene Therapy 523, 668 CD40L, X-HIGM, Gene Editing 316 Cell Processing 526 Cell Therapy - Hematopoietic 235, 273, 283, 285, 432, 523, 526, 562, 689, 734, 757 Clinical Gene Therapy 235, 314, 364, 754 CRISPR/Cas9 135, 330, 564, 732, 768 DNA Viral Vectors 106 Ex-Vivo Expansion 523 Fanconi Anemia 753 Foamy Virus 1 Gene Correction/Modification/Targeting 37, 42, 330, 432, 558, 562, 732, 753 Genetic Diseases 32, 291, 558, 753 Genetic Modification 734 Genomic Deletions 135 Hematologic Diseases 4, 42, 221, 228, 229, 231, 281, 508, 759 HIV Stem Cell Gene Therapy 333 HSC Engraftment, Hematologic Disease 732 Immunotherapy 508, 768 Infectious Diseases 333, 429, 687, 767, 768 Lentivirus Vectors 4, 135, 221, 222, 228, 229, 281, 283, 291, 333, 364, 458, 530, 536, 564, 754, 759 Long Term Expression 429, 689 Luekocyte Adhesion Deficiency 285 Mathematical Modeling 314 Mixed Chimerism 231 Nanobiotechnology 536 Platelets 668 Retrovirus Vectors 530, 687 RNA Viral Vectors 1 TALEN 562 TALENs 316, 758 Targeted Gene Expression 668 Toxicology Studies 32 Vector Characterization/Biology 1 Vector Production 458 Viral Transduction Enhancers 536 Zinc-Finger Nucleases 37, 734 β-Thalassemias, Hemoglobinopathies 222
Molecular Therapy Volume 24, Supplement 1, May 2016 Copyright © The American Society of Gene & Cell Therapy
Hemophilia AAV Vectors
52, 78, 92, 223, 227, 253, 296, 456, 545, 550, 684, 688, 760 Animal Models 52, 223, 227, 296, 492, 756, 760 Assay Development 171 Chimeric Antigen Receptors 434 Clinical Gene Therapy 171 CRISPR/Cas9 128 DNA Viral Vectors 253 Gene Correction/Modification/Targeting 128 Gene Expression 5, 171, 456, 545 Hematologic Diseases 227, 760 Immunotherapy 434, 688 Insulin Therapy 550 Lentivirus Vectors 5, 756 Non-Viral Gene Delivery 492 Oversized Vector 92 Pharmacology/Toxicology 550 Regulatory T Cell 434 Transgene Engineering 456 Ultrasound 492 Vector Immunology/Host Responses 78 Vector Production 92, 684
High Quality Grade AAV Vectors Clinical Gene Therapy Good Manufacturing Procedures
592 592 592
HIV Latency CRISPR/Cas9 125 Infectious Diseases 125 Targeted Gene Expression 125 HIV Stem Cell Gene Therapy Hematopoietic Stem Cells Infectious Diseases Lentivirus Vectors HIV
333 333 333
AAV Vectors 44 Animal Models 427 Cell Therapy - Hematopoietic 427, 763 Cell Therapy - Other 763 Gene Correction/Modification/Targeting 44 Hematologic Diseases 763 Immunotherapy 427 Infectious Diseases 44
HIV-1, Virual Escape CRISPR/Cas9 331 Infectious Diseases 331 Lentivirus Vectors 331 Homology-Directed Repair CRISPR/Cas9 568 Gene Correction/Modification/Targeting 568 Genetic Diseases 568 HSC Engraftment, Hematologic Disease CRISPR/Cas9 732 Gene Correction/Modification/Targeting 732 Hematopoietic Stem Cells 732 S335
KEYWORD INDEX HSV Vectors AAV Vectors 94 Animal Models 656, 660, 667 Cancer - Gene Therapy 184, 520, 658, 664, 667, 680 Cancer - Targeted Therapy 658, 660, 665, 666 Chronic Pain 111 Colorectal Cancer 664 DNA Viral Vectors 107, 520, 664 Gene Therapy 94 Glioma 665 Immunotherapy 518, 666 Neurological Disorders 111, 114, 184, 657, 665 Non-Viral Gene Delivery 680 Oncolytic Vectors 520 Oncolytic Viruses 666 Retrovirus Vectors 657 Targeted Gene Expression 111, 114 Therapeutic Gene Modulation 656 Vector Characterization/Biology 107 Vector Immunology/Host Responses 518 Vector Production 94 Human Immune Response to AAV AAV Vectors Clinical Gene Therapy Long Term Expression
24 24 24
Humoral Immune Response AAV Vectors Vector Characterization/Biology Vector Immunology/Host Responses
82 82 82
Hybrid Vector AAV Vectors Cancer - Gene Therapy Vector Production
672 672 672
IL13Ra2, GBM, solid tumors Cancer - T-Cell Therapy 76 Immunotherapy 76 Targeted Gene Expression 76 IL-4
Cancer - T-Cell Therapy 751 Chimeric Antigen Receptors 751 Immunotherapy 751
Immunodeficiency Gene Correction/Modification/Targeting 325 Genetic Diseases 325 TALENs 325 Immunotherapy AAV Vectors 81, 196, 472, 555, 688, 699 Adenovirus Cytokine 405 Adenovirus Vectors 408, 410, 414, 418, 509, 513, 516, 639, 642, 662 Animal Models 63, 64, 275, 427, 693, 740, 749, 750 Cancer - Cell Therapy 199, 276, 422, 469, 476, 511, 646, 652 Cancer - Gene Therapy 61, 142, 201, 206, 261, 405, 414, 418, 495, 509, 516, 643, 644, 653, 662, 746 Cancer - Targeted Therapy 202, 401, 408, 423, 505, 639, 647, 663, 666 S336
Cancer - T-Cell Therapy 71, 73, 75, 76, 190, 191, 193, 194, 195, 197, 198, 199, 202, 203, 206, 232, 272, 275, 277, 278, 287, 391, 405, 406, 410, 451, 455, 505, 507, 510, 512, 513, 637, 638, 640, 646, 648, 649, 651, 652, 653, 746, 751 CAR T Cells; Lymphoma; Manufacturing 287 CD19, Sleeping Beauty 278 Cell Delivery 511 Cell Processing 287, 425, 455 Cell Therapy - Hematopoietic 239, 427, 685 Cell Therapy - Other 426 Checkpoint Inhibitor 513 Chemical Conjugates 225 Chimeric Antigen Receptors 73, 190, 193, 195, 198, 203, 274, 277, 399, 406, 434, 451, 507, 510, 512, 640, 649, 651, 750, 751, 761 Clinical Gene Therapy 61, 272 Clonality, T-Cell Repertoire 232 CRISPR/Cas9 274, 422, 768 DNA Vaccines, Electroporation 401 DNA Viral Vectors 194 Gene Correction/Modification/Targeting 132, 391, 761 Gene Expression 64, 142, 749 Genetic Diseases 232, 425 Glycobiology 510 Good Manufacturing Procedures 75, 203 Hematologic Diseases 71, 276, 508, 637, 663 Hematopoietic Stem Cells 508, 768 Hemophilia 434, 688 HIV 427 HSV Vectors 518, 666 IL13Ra2, GBM, Solid Tumors 76 IL-4 751 Infectious Diseases 264, 426, 428, 433, 555, 685, 699, 768 Inflammatory Toxicity 197 Integrase Defective Lentivirus Quantification 469 Lentiviral Vectors 195 Lentivirus Vectors 51, 225, 469, 476, 638, 647, 648 Long Term Expression 693 Mutated Neoantigens 391 Non-Viral Gene Delivery 264, 278, 433, 495, 644 Oligonucleotide Based Therapies 261, 740 Oncolytic Viruses 423, 666 Pancreatic Cancer 639 Passive Immunization 699 Pediatric Cancers - Solid Tumors 649 Pharmacology/Toxicology 472, 476 Re-Administration 472 Regulatory T Cell 434 Retroviral Replicating Vector 61 RNA Viral Vectors 201, 423 Sleeping Beauty Transposition ; Minicircle 73 Synthetic Gene Delivery Systems 428 TALENs 132 Targeted Gene Expression 76 T-Cell Development, Infections, Notch, Thymus 425 T-Cell Populations 693 T-Cells 225 Telomerase 276 Tissue Engineering 239 Toxicology Studies 399 Tumor Microenvironment 662 Molecular Therapy Volume 24, Supplement 1, May 2016 Copyright © The American Society of Gene & Cell Therapy
KEYWORD INDEX Vaccines 191, 196, 395, 642, 643 Vector Immunology/Host Responses 51, 63, 81, 395, 518 WT1 193 In Vivo T Cell Engineering Cancer - Gene Therapy Cancer - T-Cell Therapy RNA Viral Vectors Induced Pluripotent Stem Cells AAV Vectors Cell Therapy - ES Cells and iPS Cells CRISPR/Cas9 Gene Correction/Modification/Targeting Hematologic Diseases Lentivirus Vectors Musculo-Skeletal Disorders Ophthalmic Diseases Targeted Gene Expression Tissue Engineering
506 506 506 553, 556 241, 243, 444 318, 557, 626, 735 243 444 241 735 553, 556 557, 626 318
Infectious Diseases AAV Vectors 44, 79, 145, 539, 555, 699 Adenovirus Vectors 18, 22, 103 Animal Models 145, 173, 431, 437, 767 Antibody Delivery 429 Antisense Oligonucleotides 586 Cancer - Targeted Therapy 103 CD8+ T Cells, Ovalbumin 79 Cell Therapy - Hematopoietic 685 Cell Therapy - Other 426 Clinical Gene Therapy 258 CRISPR/Cas9 125, 331, 574, 768 Gene Correction/Modification/Targeting 18, 44, 765 Gene Expression 681 HBV, AAV 260 HCV, AAV 258 Hematopoietic Stem Cells 333, 429, 687, 767, 768 HIV 44 HIV Latency 125 HIV Stem Cell Gene Therapy 333 HIV-1, Virual Escape 331 Immunotherapy 264, 426, 428, 433, 555, 685, 699, 768 Insertional Mutagenesis 681 Lentivirus Vectors 331, 333, 431, 574 Long Term Expression 260, 429 Non-Viral Gene Delivery 173, 264, 433, 435 Non-Viral Vectorology 479 Oligonucleotide Based Therapies 440, 586 Passive Immunization 699 Pneumonia, Sepsis, Lung Contusion 173 Respiratory/Lung Disorder 22 Retrovirus Vectors 687 RNA Viral Vectors 681 RNAi and shRNA 145, 258, 260 Synthetic Gene Delivery Systems 428 Targeted Gene Expression 125 Vaccines 22, 435, 440, 479 Vector Immunology/Host Responses 79, 437 Vector Production 539 Zinc-Finger Nucleases 765 Molecular Therapy Volume 24, Supplement 1, May 2016 Copyright © The American Society of Gene & Cell Therapy
Inflammation Gene Correction/Modification/Targeting Gene Expression Genetic Diseases Lentivirus Vectors MicroRNA Oligonucleotide Based Therapies
226 146 226 226 146 146
Inflammatory Toxicity Cancer - T-Cell Therapy 197 Immunotherapy 197 Injectable Biologic Animal Models Neurological Disorders Targeted Gene Expression Insertional Mutagenesis Animal Models Gene Expression Infectious Diseases Lentivirus Vectors RNA Viral Vectors Vector Characterization/Biology
26 26 26 2 681 681 2 681 2
Insulin Therapy AAV Vectors 550 Hemophilia 550 Pharmacology/Toxicology 550 Integrase Defective Lentivirus Quantification Cancer - Cell Therapy 469 Immunotherapy 469 Lentivirus Vectors 469 Integration sites Clinical Gene Therapy Lentivirus Vectors Retrovirus Vectors
531 531 531
Intrathecal Delivery AAV Vectors Neurological Disorders
724 724
Iron-Overload AAV Vectors MicroRNA
220 220
Keloid Adenovirus Vectors Clinical Gene Therapy Gene Expression
112 112 112
Krabbe Disease AAV Vectors Genetic Diseases Neurological Disorders
57 57 57
Lentiviral Vectors AAV Vectors 322 Cancer - T-Cell Therapy 195 Chimeric Antigen Receptors 195 CRISPR/Cas9 322
S337
KEYWORD INDEX Immunotherapy 195 Musculo-Skeletal Disorders 322 Lentivirus Vector Cell Therapy - ES Cells and iPS Cells Cell Therapy - Hematopoietic Long Term Expression
234 234 234
Lentivirus Vectors AAV Vectors 129, 305, 632, 728 Adrenoleukodystrophy 250 Animal Models 2, 431, 631, 674, 756 Bone Defect 631 Cancer - Cell Therapy 212, 462, 469, 476 Cancer - Gene Therapy 214, 217, 475, 703 Cancer - Targeted Therapy 70, 217, 647, 674 Cancer - T-Cell Therapy 121, 404, 638, 648 Cell Processing 707 Cell Reprogramming 704 Cell Therapy - ES Cells and iPS Cells 241 Cell Therapy - Hematopoietic 283, 766 Cell Therapy - Mesenchymal 632 Cell Therapy - Muscle 240 Chemical Conjugates 225 Chimeric Antigen Receptors 407 Clinical Gene Therapy 250, 279, 364, 531, 534, 711, 754 CRISPR/Cas9 3, 126, 134, 135, 327, 331, 404, 532, 564, 574 Cystic Fibrosis 711 Design of Experiment 708 Gen Expression 704 Gene Correction/Modification/Targeting 3, 121, 126, 127, 129, 130, 134, 226, 286, 533, 538, 566, 682, 764 Gene Expression 5, 609, 728, 766 Genetic Diseases 226, 291, 534, 631 Genomic Deletions 135 Hematologic Diseases 4, 221, 228, 229, 230, 279, 281, 407, 682, 686, 690, 759 Hematopoietic Stem Cells 4, 135, 221, 222, 228, 229, 281, 283, 291, 333, 364, 458, 530, 536, 564, 754, 759 Hemophilia 5, 756 HIV Stem Cell Gene Therapy 333 HIV-1, Virual Escape 331 Immunotherapy 51, 225, 469, 476, 638, 647, 648 Induced Pluripotent Stem Cells 241 Infectious Diseases 331, 333, 431, 574 Inflammation 226 Insertional Mutagenesis 2 Integrase Defective Lentivirus Quantification 469 Integration Sites 531 Long Term Expression 305, 609 Macrophages, HSC 766 Mesothelioma 217 MicroRNA 589 Musculo-Skeletal Disorders 502 Nanobiotechnology 536 Neurological Disorders 250, 327 Non-Viral Vectorology 533 Ophthalmic Diseases 33 Osteoporosis, Alpha-1 Antitrypsin 632 Pharmacology/Toxicology 476, 690 Regulatory Issues 464 Respiratory/Lung Disorders 212 S338
Retrovirus Vectors 530, 531 RNA Delivery 538 RNA Viral Vectors 288, 462, 703 RNAi and shRNA 70, 532, 589 Somatic-Transgenic Technology 305 TALENs 686 Targeted Gene Expression 214 T-Cells 225 Tissue Engineering 240, 502 Toxicology studies 33, 475 Vector Characterization/Biology 2, 230, 457, 709 Vector Immunology/Host Responses 51, 764 Vector Production 286, 288, 457, 458, 464, 703, 704, 707, 708, 709, 711 Viral Transduction Enhancers 536 Zinc-Finger Nucleases 127, 130, 566 β-Thalassemias, Hemoglobinopathies 222 Lentivirus, DMD Cell Therapy - ES Cells and iPS Cells 565 Cell Therapy - Muscle 565 TALENs 565 Leukemia and Lymphoma Cancer - T-Cell Therapy Chimeric Antigen Receptors
69, 210 69, 210
Liver Diseases Animal Models Genetic Diseases Respiratory/Lung Disorders
183 183 183
Liver AAV Vectors 120 CRISPR/Cas9 120 Gene Correction/Modification/Targeting 120 Long Term Expression AAV Vectors 24, 50, 60, 305, 390, 424 Adenovirus Vectors 324, 598 Animal Models 50, 693 Antibody Delivery 429 Butyrylcholinesterase 60 Cancer - Gene Therapy 598 Cardiovascular Diseases 172 Cell Therapy - ES Cells and iPS Cells 234 Cell Therapy - Hematopoietic 234, 689 Cell Therapy - Other 161 Clinical Gene Therapy 24 Diabetes 50 Gene Expression 60, 172, 324, 609 Genetic Diseases 161, 439 HBV, AAV 260 Hematopoietic Stem Cells 429, 689 Human Immune Response to AAV 24 Immunotherapy 693 Infectious Diseases 260, 429 Lentivirus Vector 234 Lentivirus Vectors 305, 609 Neuromuscular Disorders 390 Non-Viral Gene Delivery 439 RNAi and shRNA 260 Somatic-Transgenic Technology 305 Molecular Therapy Volume 24, Supplement 1, May 2016 Copyright © The American Society of Gene & Cell Therapy
KEYWORD INDEX T-Cell Populations Vector Immunology/Host Responses Luekocyte Adhesion Deficiency AAV Vectors Cell Therapy - Hematopoietic Hematopoietic Stem Cells
693 424
Clinical Gene Therapy Lentivirus Vectors
661 217
285 285 285
Metabolic Disease AAV Vectors Animal Models Neurological Disorders
356 356 356
Lung Cancer Nanobiotechnology 489 Non-Viral Gene Delivery 489 RNAi and shRNA 489 Lysosomal Storage Deseases, Enzyme Replacement Animal Models Cell Therapy - Other Metabolic Diseases
430 430 430
Lysosomal Storage Disease AAV Vectors Genetic Diseases Neurological Disorders
348 348 348
Lysosomal Storage Diseases AAV Vectors Animal Models Neurological Disorders
361 361 361
Macrophages, HSC Cell Therapy - Hematopoietic Gene Expression Lentivirus Vectors
766 766 766
Magnetic Targeting, MRI Cell Therapy - Other MicroRNA Non-Viral Gene Delivery
338 338 338
Mathematical Modeling Clinical Gene Therapy Hematopoietic Stem Cells
314 314
Measles Virus, Glioblastoma Cancer - Gene Therapy MicroRNA RNA Viral Vectors
62 62 62
Meganuclease, AAV Vectors Cancer - T-Cell Therapy Chimeric Antigen Receptors Gene Correction/Modification/Targeting
394 394 394
Meganucleases Cancer - T-Cell Therapy Chimeric Antigen Receptors Gene Correction/Modification/Targeting Genetic Diseases Neuromuscular Disorders
200 200 138, 579 579 138
Mesothelioma Adenovirus Vectors 211, 661 Apoptosis 211 Cancer - Gene Therapy 217 Cancer - Targeted Therapy 211, 217, 661 Molecular Therapy Volume 24, Supplement 1, May 2016 Copyright © The American Society of Gene & Cell Therapy
Metabolic Diseases AAV 25 AAV Vectors 48, 58, 86, 93, 157, 158, 159, 162, 168, 218, 346, 349, 351, 352, 359, 366, 372, 382, 468, 611, 696 Adenovirus Vectors 47, 179, 484 Animal Models 166, 168, 346, 357, 430, 477 Cardiovascular Diseases 179, 358 Cell Therapy - ES Cells and iPS Cells 347 Cell Therapy - Hematopoietic 163 Cell Therapy - Mesenchymal 354 Cell Therapy - Other 430 Clinical Gene Therapy 353 Diabetes 354 Gastrointestinal Inflammation 477 Gene Correction/Modification/Targeting 48, 86, 93, 347, 354, 359 Gene Expression 158, 159, 477 Genetic Diseases 25, 46, 47, 160, 163, 166, 169, 353, 357, 358, 360, 369 Lysosomal Storage Deseases, Enzyme Replacement 430 Methylmalonic Acidemia 168 MitoTimer 358 MPS1 93 mRNA 170 Musculo-Skeletal Disorders 382 Neurological Disorders 25, 58, 157, 160, 162, 349, 351, 352, 360, 366, 369, 372, 611 Non-Viral Gene Delivery 169, 355 Ophthalmic Diseases 46 Promoterless Vector 48 RNA Delivery 170 SERCaMP, ATV6, ER Stress 218 Synthetic Gene Delivery Systems 170 TLR9 Signaling 696 Toxicology Studies 468 Urea Cycle Disorder 347 Vector Characterization/Biology 218, 355 Vector Immunology/Host Responses 696 Zinc-Finger Nucleases 484 Methylmalonic Acidemia AAV Vectors Animal Models Metabolic Diseases
168 168 168
MicroRNA AAV Vectors 220, 379, 606, 669, 737, 739 Amyotrophic Lateral Sclerosis 606 Animal Models 529, 738 Cancer - Gene Therapy 62, 144, 154, 587, 669 Cancer - Targeted Therapy 67 Cardiovascular Diseases 19 Cell Processing 245 Cell Therapy - Mesenchymal 245 S339
KEYWORD INDEX Cell Therapy - Other 338 Clinical Gene Therapy 282 Familial Hypercholesterolaemia 19 Gene Correction/Modification/Targeting 742 Gene Expression 146 Genetic Diseases 606 HCV, AAV 143 Inflammation 146 Iron-Overload 220 Lentivirus Vectors 589 Magnetic Targeting, MRI 338 Measles Virus, Glioblastoma 62 Musculo-Skeletal Disorders 379 Neurological Disorders 738, 742 Non-Viral Gene Delivery 19, 338 Oligonucleotide Based Therapies 146 Oncolytic Viruses 67 Ophthalmic Diseases 739 Prostate Cancer 669 Regulatory Issues 143 RNA Viral Vectors 62, 67, 529 RNAi and shRNA 143, 144, 154, 587, 589, 737 Targeted Gene Expression 282
Muscular Dystrophy AAV Vectors Gene Expression Musculo-Skeletal Disorders
726 726 726
93 93 93
Musculo-Skeletal Disorders AAV 499 AAV Vectors 49, 322, 378, 379, 380, 381, 382, 387, 480, 483, 497, 503, 552, 622, 624, 629, 630, 722, 726 Adenovirus Vectors 104 Animal Model 629 Animal Models 388, 499, 503, 633, 635 Cancer - Cell Therapy 636 Cell Reprogramming 525 Cell Therapy - ES Cells and iPS Cells 312 Cell Therapy - Mesenchymal 238 Cell Therapy - Muscle 238, 446, 449, 525, 577, 635, 636 Cell Therapy - Other 446, 454 Clinical Gene Therapy 328, 387, 480, 497 CRISPR/Cas9 104, 139, 317, 322, 385, 501, 504, 577, 735 DNA Viral Vectors 630 Epigenetic regulation 385 Exon Skipping 624 FSHD 153 Gene Correction/Modification/Targeting 139, 312, 317, 483, 501, 504 Gene Expression 449, 454, 499, 622, 726 Genetic Diseases 49, 381, 741 Induced Pluripotent Stem Cells 735 Lentiviral Vectors 322 Lentivirus Vectors 502 Metabolic Diseases 382 MicroRNA 379 Muscular Dystrophy 726 Neurological Disorders 741 Neuromuscular Disorders 380, 388, 500, 552, 629, 633 Non-Invasive 633 Oligonucleotide Based Therapies 153, 328, 741 Pompe Disease 49, 381 RNAi and shRNA 386, 500, 625 Spinal Muscular Atrophy, Type 1 480 Sporadic Inclusion Body Myositis, Follistatin 497 TALENs 139 Targeted Gene Expression 153 Tissue Engineering 502 Toxicology Studies 386, 625 Vaccines 722 Vector Immunology/Host Responses 378
mRNA Metabolic Diseases RNA Delivery Synthetic Gene Delivery Systems
170 170 170
Mutated Neoantigens Cancer - T-Cell Therapy 391 Gene Correction/Modification/Targeting 391 Immunotherapy 391
Mucopolysaccharidosis AAV Vectors Gene Correction/Modification/Targeting Zinc-Finger Nucleases
485 485 485
Multiple Myeloma Cancer - Gene Therapy Cancer - T-Cell Therapy Zinc-Finger Nucleases
752 752 752
Minicircle AAV Vectors DNA Viral Vectors Gene Expression Non-Viral Vectorology Ophthalmic Diseases Respiratory/Lung Disorders
306 306 743 743 306 743
Mitochondrial biogenesis Cancer - Targeted Therapy Hematologic Diseases
65 65
MitoTimer Cardiovascular Diseases Genetic Diseases Metabolic Diseases
358 358 358
Mixed Chimerism Hematologic Diseases Hematopoietic Stem Cells
231 231
MPS1 AAV Vectors Gene Correction/Modification/Targeting Metabolic Diseases
S340
Nanobiotechnology Cancer - Cell Therapy Cancer - Targeted Therapy Cancer - T-Cell Therapy Cell Delivery Chemical Conjugates Ethical Issues Gene Correction/Modification/Targeting
209, 442 209, 396, 494 396 442 342 720 563, 720
Molecular Therapy Volume 24, Supplement 1, May 2016 Copyright © The American Society of Gene & Cell Therapy
KEYWORD INDEX Hematopoietic Stem Cells 536 Lentivirus Vectors 536 Lung Cancer 489 Non-Viral Gene Delivery 339, 489, 494 Ophthalmic Diseases 339, 342 Polymeric, PBAE 494 Regulatory Issues 600 RNAi and shRNA 489 Stem Cells; Ovarian Cancer; Drug Delivery 209 Targeted Gene Therapy 339 Vaccines 600 Viral Transduction Enhancers 536 Zinc-Finger Nucleases 563 Natural History and Trial Development AAV Vectors Clinical Gene Therapy Neuromuscular Disorders
715 715 715
Neural Stem Cells, Pediatric Brain Tumors Cancer - Targeted Therapy Cell Therapy - Neuronal
443 443
Neuritin AAV Vectors Neurological Disorders
371 371
Neurological Disorders AAV 25 AAV Vectors 27, 30, 45, 54, 55, 56, 57, 58, 59, 157, 162, 186, 252, 255, 256, 271, 295, 301, 321, 337, 348, 349, 351, 352, 356, 361, 362, 366, 368, 370, 371, 372, 376, 540, 583, 604, 605, 610, 611, 612, 613, 616, 618, 619, 621, 724 Adrenoleukodystrophy 250 Aging 619 Alzheimer’s Disease 59, 567 Animal Models 26, 54, 55, 56, 186, 256, 337, 356, 361, 362, 376, 604, 612, 613, 619, 627, 738 Axon Regeneration 271 Brain Delivery 618 Cancer - Cell Therapy 528 Cancer - Gene Therapy 184 Cancer - Targeted Therapy 665 Capsid engineering 252 Cell Therapy - ES Cells and iPS Cells 528 Cell Therapy - Hematopoietic 374 Chronic Pain 111 Clinical Gene Therapy 250, 267, 367 CRISPR/Cas9 321, 327, 567 Down Syndrome 53 Enteric Nervous System, Gastrointestinal Disorders 612 Ethical Issues 627 Gene Correction/Modification/Targeting 295, 567, 605, 742 Gene Expression 252, 370, 373, 610, 616, 618, 736 Genetic Diseases 25, 27, 45, 53, 57, 160, 348, 360, 363, 369, 373, 621, 741 Glioma 665 GM2 Gangliosidosis 363 HSV Vectors 111, 114, 184, 657, 665 Injectable Biologic 26 Intrathecal Delivery 724 Krabbe Disease 57 Molecular Therapy Volume 24, Supplement 1, May 2016 Copyright © The American Society of Gene & Cell Therapy
Lentivirus Vectors 250, 327 Lysosomal Storage Disease 348 Lysosomal Storage Diseases 361 Metabolic Disease 356 Metabolic Diseases 25, 58, 157, 160, 162, 349, 351, 352, 360, 366, 369, 372, 611 MicroRNA 738, 742 Musculo-Skeletal Disorders 741 Neuritin 371 Neuromuscular Disorders 259 Neurotrophic Factor, Parkinson 610 Novel Capsid 256 Oligonucleotide Based Therapies 259, 741 Ophthalmic Diseases 267, 736 Optogenetics 186 Pharmacology/Toxicology 30, 368 Pitx3, Mitochondria, Antioxidants, Transcription 376 Retrovirus Vectors 367, 657 RNA Viral Vectors 374 RNAi and shRNA 53, 583, 614 Synthetic Gene Delivery Systems 614 Targeted Gene Expression 26, 111, 114 U1 Adaptors, Parkinsons, Huntingtons 259 Urea Cycle Disorder 45 Vector Characterization/Biology 255, 301, 540 Neuromuscular Disorders AAV Vectors 85, 90, 377, 380, 389, 390, 498, 551, 552, 629, 715, 721 Animal Model 629 Animal Models 377, 388, 498, 623, 633, 721 Cell Delivery 584 Cell Therapy - ES Cells and iPS Cells 730 Cell Therapy - Muscle 85 Clinical Gene Therapy 715 CRISPR/Cas9 730 DMD Dogs 721 Duchenne Muscular Dystrophy; Exon Skipping 584 Evolution and Development 389 Gene Correction/Modification/Targeting 138, 730 Genetic Diseases 150, 551 Long Term Expression 390 Meganucleases 138 Musculo-Skeletal Disorders 380, 388, 500, 552, 629, 633 Natural History and Trial Development 715 Neurological Disorders 259 Non-Invasive 633 Oligonucleotide Based Therapies 150, 259, 584, 623 RNAi and shRNA 500 U1 Adaptors, Parkinsons, Huntingtons 259 Vector Characterization/Biology 389 Vector Immunology/Host Responses 90 Neurotrophic Factor, Parkinson AAV Vectors Gene Expression Neurological Disorders
610 610 610
Non-Invasive Animal Models Musculo-Skeletal Disorders Neuromuscular Disorders
633 633 633 S341
KEYWORD INDEX Non-Small Cell Lung Cancer Cancer - Gene Therapy Cell Therapy - Lung RNAi and shRNA
585 585 585
Non-Viral Gene Delivery Animal Models 173, 490, 492 Bioprocess 343 Cancer - Gene Therapy 152, 155, 493, 495, 593, 599, 644, 680 Cancer - Targeted Therapy 72, 155, 494 Cancer - T-Cell Therapy 278 Cardiovascular Diseases 19 Cd19, Sleeping Beauty 278 Cell Processing 343 Cell Reprogramming 343, 344 Cell Therapy - ES Cells and iPS Cells 628 Cell Therapy - Muscle 628 Cell Therapy - Other 338 CGD Stem Cell Gene Correction 41 Chimeric Antigen Receptors 705 Clinical Gene Therapy 72 CRISPR/Cas9 41, 335, 487 DNA Vaccines 436 Exosomes 341 Familial Hypercholesterolaemia 19 Gene Correction/Modification/Targeting 41, 487, 601 Genetic Diseases 169, 439, 597, 601 Hemophilia 492 HSV Vectors 680 Immunotherapy 264, 278, 433, 495, 644 Infectious Diseases 173, 264, 433, 435 Long Term Expression 439 Lung Cancer 489 Magnetic Targeting, MRI 338 Metabolic Diseases 169, 355 MicroRNA 19, 338 Nanobiotechnology 339, 489, 494 Non-Viral Vectorology 599 Ophthalmic Diseases 339 PDGF, Angiogenesis 152 Physcial Delviery 491 Pneumonia, Sepsis, Lung Contusion 173 Polymeric, PBAE 494 Polymers 493 Respiratory/Lung Disorders 490, 597 RNA Delivery 335, 341, 593 RNAi and shRNA 152, 489, 599 Synthetic Gene Delivery Systems 493 Targeted Gene Therapy 339 Tissue Engineering 491 Transposon 628, 705 Ultrasound 492 Vaccines 435, 436, 491 Vector Characterization/Biology 344, 355 Non-Viral Vectorology Cancer - Gene Therapy Cancer - T-Cell Therapy Gene Correction/Modification/Targeting Gene Expression Hematologic Diseases S342
599 213 533 289, 743 213
Infectious Diseases 479 Lentivirus Vectors 533 Minicircle 743 Non-Viral Gene Delivery 599 Respiratory/Lung Disorders 743 RNAi and shRNA 599 Vaccines 479 Vector Characterization/Biology 289 Notch Signaling Animal Models Cardiovascular Diseases Cell Therapy - Other
447 447 447
Novel Capsid AAV Vectors Animal Models Neurological Disorders
256 256 256
Novel Molecules to Enhance Viral Entry Adenovirus Vectors 176 Pharmacology/Toxicology 176 Respiratory/Lung Disorders 176 Oligonucleotide Based Therapies Animal Models 580, 623, 740 Antisense Oligonucleotides 586 Aptamers 148, 744 Bioinformatics 148 Cancer - Cell Therapy 263 Cancer - Gene Therapy 261 Cancer - Targeted Therapy 262, 590 Cell Delivery 584 Clinical Gene Therapy 328 Duchenne Muscular Dystrophy; Exon Skipping 584 FSHD 153 Gene Expression 146 Genetic Diseases 150, 581, 741 Hematologic Diseases 581 Immunotherapy 261, 740 Infectious Diseases 440, 586 Inflammation 146 MicroRNA 146 Musculo-Skeletal Disorders 153, 328, 741 Neurological Disorders 259, 741 Neuromuscular Disorders 150, 259, 584, 623 QC/QA Assays 263 Respiratory/Lung Disorders 580 RNA Delivery 148, 262, 744 Splice, X-Linked Agammaglobulinemia 581 Synthetic Gene Delivery Systems 590 Targeted Gene Expression 153 U1 Adaptors, iRGD, KRAS, MYC, Pancreatic Cancer 262 U1 Adaptors, Parkinsons, Huntingtons 259 Vaccines 440 Oncolytic Adenovirus Cancer - Cell Therapy Cancer - T-Cell Therapy Chimeric Antigen Receptors
515 515 515
Molecular Therapy Volume 24, Supplement 1, May 2016 Copyright © The American Society of Gene & Cell Therapy
KEYWORD INDEX Oncolytic Vectors Cancer - Gene Therapy DNA Viral Vectors Hsv Vectors
520 520 520
Oncolytic Virus Animal Models 411 Apoptosis 411 Cancer - Targeted Therapy 411 Cancer - T-Cell Therapy 68 RNA Viral Vectors 68 Vaccines 68 Oncolytic Viruses Cancer - Targeted Therapy HSV Vectors Immunotherapy MicroRNA RNA Viral Vectors
67, 423, 654, 666 666 423, 666 67 67, 423, 654
Ophthalmic Diseases AAV Vectors 28, 29, 31, 80, 83, 88, 96, 98, 188, 189, 248, 249, 265, 268, 270, 306, 544, 553, 556, 607, 608, 617, 620, 739 Age-Related Macular Degeneration 189 Animal Models 96, 189 Anti-Transgene 694 Cell Therapy - Other 445 Cellular Clearance 187 Chemical Conjugates 342 Clinical Gene Therapy 83, 248, 249, 267 CRISPR/Cas9 124, 329 Delivery Techniques 29 DNA Viral Vectors 306 Exosomes 594 Functional MRI (fMRI), Visual Pathways, Brain 265 Gene Correction/Modification/Targeting 124, 188, 329, 620 Gene Expression 98, 187, 270, 320, 608, 736 Genetic Diseases 28, 46, 83, 265, 617 Glaucoma 188 Good Manufacturing Procedures 445 Induced Pluripotent Stem Cells 553, 556 Lentivirus Vectors 33 Metabolic Diseases 46 MicroRNA 739 Minicircle 306 Nanobiotechnology 339, 342 Neurological Disorders 267, 736 Non-Viral Gene Delivery 339 Optogenetic Therapy 270 Pharmacology/Toxicology 31, 88, 268 Retina 544 Targeted Gene Expression 29, 320 Targeted Gene Therapy 339 Toxicology Studies 33 Vector Characterization/Biology 594, 607 Vector Immunology/Host Responses 80, 694 Vector Transduction 594 Wet Age-Related Macular Degeneration 248
Molecular Therapy Volume 24, Supplement 1, May 2016 Copyright © The American Society of Gene & Cell Therapy
Optogenetic Therapy AAV Vectors Gene Expression Ophthalmic Diseases
270 270 270
Optogenetics AAV Vectors Animal Models Neurological Disorders
186 186 186
Orbital Shaken Bioreactors AAV Vectors Good Manufacturing Procedures Vector Production
463 463 463
Osteoporosis, Alpha-1 Antitrypsin AAV Vectors Cell Therapy - Mesenchymal Lentivirus Vectors
632 632 632
Oversized Vector AAV Vectors 92 Hemophilia 92 Vector Production 92 Pancreatic Cancer Adenovirus Vectors 639 Cancer - Targeted Therapy 639 Immunotherapy 639 Passive Immunization AAV Vectors 699 Immunotherapy 699 Infectious Diseases 699 PD-1 Cancer - T-Cell Therapy 572 CRISPR/Cas9 572 Gene Correction/Modification/Targeting 572 PDGF, Angiogenesis Cancer - Gene Therapy Non-Viral Gene Delivery RNAi and shRNA
152 152 152
Pediatric Cancers - Solid Tumors Cancer - T-Cell Therapy 649 Chimeric Antigen Receptors 649 Immunotherapy 649 Pharmacology/Toxicology AAV Vectors 30, 31, 88, 268, 299, 365, 368, 472, 550 Adenovirus Vectors 176 Aptamers 671 Cancer - Cell Therapy 476 Cancer - Targeted Therapy 671 Cell Processing 375 Cell Therapy - Neuronal 375 Gene Expression 299 GPCR, TDP-43, Cannabinoids 375 Hematologic Diseases 690 Hemophilia 550 Immunotherapy 472, 476 Insulin Therapy 550 S343
KEYWORD INDEX Lentivirus Vectors 476, 690 Neurological Disorders 30, 368 Novel Molecules to Enhance Viral Entry 176 Ophthalmic Diseases 31, 88, 268 Re-Administration 472 Respiratory/Lung Disorders 176 Toxicology Studies 365 Physcial Delviery Non-Viral Gene Delivery 491 Tissue Engineering 491 Vaccines 491 Pitx3, Mitochondria, Antioxidants, Transcription AAV Vectors Animal Models Neurological Disorders
376 376 376
Platelets Cancer - Gene Therapy Hematopoietic Stem Cells Targeted Gene Expression
668 668 668
Pneumonia, Sepsis, Lung Contusion Animal Models Infectious Diseases Non-Viral Gene Delivery
173 173 173
Polymeric, PBAE Cancer - Targeted Therapy 494 Nanobiotechnology 494 Non-Viral Gene Delivery 494 Polymers Cancer - Gene Therapy Non-Viral Gene Delivery Synthetic Gene Delivery Systems Pompe Disease AAV Vectors Genetic Diseases Musculo-Skeletal Disorders
493 493 493 49, 381 49, 381 49, 381
Precision Medicine AAV Vectors 7 Bioinformatics 537 Clinical Gene Therapy 537 CRISPR/Cas9 141, 575 Gene Correction/Modification/Targeting 575 QC/QA Assays 141 RNA Viral Vectors 537 Single-Molecule, Real-Time Sequencing 7 Vector Characterization/Biology 7 Pre-Existing AAV Antibodies AAV Vectors Animal Models Vector Immunology/Host Responses
698 698 698
Process Modeling; Monte Carlo Simulations Cancer - T-Cell Therapy Cell Processing Chimeric Antigen Receptors
650 650 650
S344
Promoterless Vector AAV Vectors Gene Correction/Modification/Targeting Metabolic Diseases
48 48 48
Promoters AAV Vectors Cell Therapy - Neuronal Gene Expression
99 99 99
Prostate Cancer AAV Vectors Cancer - Gene Therapy MicroRNA
669 669 669
Pulmonary Macrophage Transplantation Cell Therapy - Lung Cell Therapy - Other Respiratory/Lung Disorders
35 35 35
QC/QA Assays AAV Vectors 34, 290, 467 Adenovirus Vectors 461 Cancer - Cell Therapy 263 Cancer - Gene Therapy 461 Cardiovascular Diseases 244 Cell Therapy - ES Cells and iPS Cells 244 CRISPR/Cas9 141 Crude lysate 290 Hematologic Diseases 473 Oligonucleotide Based Therapies 263 Precision Medicine 141 Thermostability 34 Vector Characterization/Biology 34, 156, 473 Vector Immunology/Host Responses 467 Vector Production 156, 290 Reactive Oxygen Species Cancer - Cell Therapy Cancer - Targeted Therapy Gene Expression
216 216 216
Re-Administration AAV Vectors 472 Immunotherapy 472 Pharmacology/Toxicology 472 Regulation AAV Vectors Gene Expression Vector Characterization/Biology
546 546 546
Regulatory Issues Cell Therapy - Other 718 Clinical Gene Therapy 719 Commercialization 718 Common Rule 717 Ethical Issues 717 HCV, AAV 143 Lentivirus Vectors 464 MicroRNA 143 Nanobiotechnology 600 Regulatory Issues 719 Molecular Therapy Volume 24, Supplement 1, May 2016 Copyright © The American Society of Gene & Cell Therapy
KEYWORD INDEX RNAi and shRNA Vaccines Vector Production
143 465, 600 464, 465
Regulatory T Cell Chimeric Antigen Receptors 434 Hemophilia 434 Immunotherapy 434 Reovirus Animal Models Cancer - Targeted Therapy Vector Immunology/Host Responses
415 415 415
Respiratory/Lung Disorder Adenovirus Vectors 22 Infectious Diseases 22 Vaccines 22 Respiratory/Lung Disorders Adenovirus Vectors 176 Animal Models 167, 182, 183, 490, 580 Cancer - Cell Therapy 212 Cell Therapy - ES Cells and iPS Cells 122 Cell Therapy - Lung 35, 182 Cell Therapy - Other 35 CRISPR/Cas9 23, 167 Gene Correction/Modification/Targeting 122 Gene Expression 743 Genetic Diseases 183, 597 Lentivirus Vectors 212 Liver Diseases 183 Minicircle 743 Non-Viral Gene Delivery 490, 597 Non-Viral Vectorology 743 Novel Molecules to Enhance Viral Entry 176 Oligonucleotide Based Therapies 580 Pharmacology/Toxicology 176 Pulmonary Macrophage Transplantation 35 RNAi and shRNA 23 Targeted Gene Expression 181 Vector Characterization/Biology 181 Retina AAV Vectors Ophthalmic Diseases
544 544
Retroviral Replicating Vector Cancer - Gene Therapy 61 Clinical Gene Therapy 61 Immunotherapy 61 Retrovirus Vectors Animal Models Cancer - Gene Therapy Chemical Conjugates Clinical Gene Therapy Gene Correction/Modification/Targeting Genetic Diseases Hematopoietic Stem Cells Hsv Vectors Infectious Diseases Integration Sites Molecular Therapy Volume 24, Supplement 1, May 2016 Copyright © The American Society of Gene & Cell Therapy
284 403, 602 602 224, 367, 531 284 224 530, 687 657 687 531
Lentivirus Vectors Neurological Disorders Vector Immunology/Host Responses
530, 531 367, 657 403
RNA Delivery Apoptosis 149 Aptamers 148, 744 Bioinformatics 148 Cancer - Gene Therapy 149, 593 Cancer - Targeted Therapy 262 CRISPR/Cas9 335 Exosomes 341 Gene Correction/Modification/Targeting 538 Lentivirus Vectors 538 Metabolic Diseases 170 mRNA 170 Non-Viral Gene Delivery 335, 341, 593 Oligonucleotide Based Therapies 148, 262, 744 Sendai Virus 149 Synthetic Gene Delivery Systems 170 U1 Adaptors, iRGD, KRAS, MYC, Pancreatic Cancer 262 RNA Viral Vectors Animal Models 205, 529, 673 Bioinformatics 537 Cancer - Cell Therapy 462 Cancer - Gene Therapy 62, 201, 205, 409, 416, 506, 673, 703 Cancer - Targeted Therapy 67, 416, 423, 654 Cancer - T-Cell Therapy 68, 506 Cell Therapy - Hematopoietic 6, 374, 683 Cell Therapy - Lung 438 Clinical Gene Therapy 537 Foamy Virus 1, 683 Gene Expression 681 Hematologic Diseases 683 Hematopoietic Stem Cells 1 Immunotherapy 201, 423 In Vivo T Cell Engineering 506 Infectious Diseases 681 Insertional Mutagenesis 681 Lentivirus Vectors 288, 462, 703 Measles Virus, Glioblastoma 62 MicroRNA 62, 67, 529 Neurological Disorders 374 Oncolytic Virus 68 Oncolytic Viruses 67, 423, 654 Precision Medicine 537 Vaccines 68 Vector Characterization/Biology 1, 409 Vector Immunology/Host Responses 438 Vector Production 6, 288, 703 RNAi and shRNA AAV Vectors 145, 583, 737 Adenovirus Vectors 105 Animal Models 145 Cancer - Gene Therapy 144, 152, 154, 585, 587, 588, 599 Cancer - Targeted Therapy 70, 105 Cell Therapy - Lung 585 Clinical Gene Therapy 258 Crispr/Cas9 23, 532 Down Syndrome 53 S345
KEYWORD INDEX Genetic Diseases 53 Hbv, AAV 260 Hcv, AAV 143, 258 Infectious Diseases 145, 258, 260 Lentivirus Vectors 70, 532, 589 Long Term Expression 260 Lung Cancer 489 MicroRNA 143, 144, 154, 587, 589, 737 Musculo-Skeletal Disorders 386, 500, 625 Nanobiotechnology 489 Neurological Disorders 53, 583, 614 Neuromuscular Disorders 500 Non-Small Cell Lung Cancer 585 Non-Viral Gene Delivery 152, 489, 599 Non-Viral Vectorology 599 Pdgf, Angiogenesis 152 Regulatory Issues 143 Respiratory/Lung Disorders 23 Synthetic Gene Delivery Systems 588, 614 Toxicology Studies 386, 625 Routes Of Administration AAV Vectors Genetic Diseases Targeted Gene Expression
300 300 300
Sendai Virus Apoptosis 149 Cancer - Gene Therapy 149 RNA Delivery 149 SERCaMP, ATV6, ER Stress AAV Vectors Metabolic Diseases Vector Characterization/Biology Single-Molecule, Real-Time Sequencing AAV Vectors Precision Medicine Vector Characterization/Biology
218 218 218 7 7 7
Sleeping Beauty Transposition ; Minicircle Cancer - T-Cell Therapy 73 Chimeric Antigen Receptors 73 Immunotherapy 73 Somatic-Transgenic Technology AAV Vectors Lentivirus Vectors Long Term Expression
305 305 305
Spinal Muscular Atrophy, Type 1 AAV Vectors Clinical Gene Therapy Musculo-Skeletal Disorders
480 480 480
Splice, X-Linked Agammaglobulinemia Genetic Diseases Hematologic Diseases Oligonucleotide Based Therapies
581 581 581
S346
Sporadic Inclusion Body Myositis, Follistatin AAV Vectors Clinical Gene Therapy Musculo-Skeletal Disorders
497 497 497
Stem Cells; Ovarian Cancer; Drug Delivery Cancer - Cell Therapy 209 Cancer - Targeted Therapy 209 Nanobiotechnology 209 Synthetic Biology CRISPR/Cas9 136 Gene Correction/Modification/Targeting 136 TALENs 136 Synthetic Gene Delivery Systems Cancer - Gene Therapy 493, 588 Cancer - Targeted Therapy 590 Immunotherapy 428 Infectious Diseases 428 Metabolic Diseases 170 mRNA 170 Neurological Disorders 614 Non-Viral Gene Delivery 493 Oligonucleotide Based Therapies 590 Polymers 493 RNA Delivery 170 RNAi and shRNA 588, 614 TALEN Cell Therapy - Hematopoietic Gene Correction/Modification/Targeting Hematopoietic Stem Cells
562 562 562
TALENs AAV Vectors 316, 569 Animal Models 758 Cancer - T-Cell Therapy 397, 676 CD40L, X-HIGM, Gene Editing 316 Cell Therapy - ES Cells and iPS Cells 332, 565 Cell Therapy - Muscle 565 Chimeric Antigen Receptors 397, 676 CRISPR/Cas 569 CRISPR/Cas9 136, 137, 139, 692 Gene Correction/Modification/Targeting 132, 136, 137, 139, 325, 332, 569 Genetic Diseases 325 Hematologic Diseases 686, 692 Hematopoietic Stem Cells 316, 758 Immunodeficiency 325 Immunotherapy 132 Lentivirus Vectors 686 Lentivirus, DMD 565 Musculo-Skeletal Disorders 139 Synthetic Biology 136 Thalassemia 692 Tandem Bispecific Chimeric Antigen Receptors Cancer - Targeted Therapy Cancer - T-Cell Therapy Chimeric Antigen Receptors
392 392 392
Molecular Therapy Volume 24, Supplement 1, May 2016 Copyright © The American Society of Gene & Cell Therapy
KEYWORD INDEX Targeted Epigenetic Editing CRISPR/Cas9 729 Epigenetic Regulation 729 Gene Correction/Modification/Targeting 729
T-Cell Populations Animal Models 693 Immunotherapy 693 Long Term Expression 693
Targeted Gene Expression AAV Vectors 29, 298, 300, 307, 478, 542, 695, 723 Animal Models 26, 675 Barcoding 542 Cancer - Cell Therapy 679 Cancer - Gene Therapy 214, 668 Cancer - Targeted Therapy 723 Cancer - T-Cell Therapy 76 Cancer Stem Cells, Tumor Microenvironment 679 Cell Reprogramming 40 Chronic Pain 111 Clinical Gene Therapy 282 Colorectal Cancer 675 CRISPR/Cas9 125, 557, 626 Delivery Techniques 29 DNA Viral Vectors 108 FSHD 153 Gene Correction/Modification/Targeting 40, 315, 542 Gene Expression 320, 675, 679 Genetic Diseases 300 Hematopoietic Stem Cells 668 HIV Latency 125 HSV Vectors 111, 114 IL13Ra2, GBM, Solid Tumors 76 Immunotherapy 76 Induced Pluripotent Stem Cells 557, 626 Infectious Diseases 125 Injectable Biologic 26 Lentivirus Vectors 214 MicroRNA 282 Musculo-Skeletal Disorders 153 Neurological Disorders 26, 111, 114 Oligonucleotide Based Therapies 153 Ophthalmic Diseases 29, 320 Platelets 668 Respiratory/Lung Disorders 181 Routes of Administration 300 Vector Characterization/Biology 108, 181, 298, 307, 478, 695 Xist-Mediated Chromosome Silencing 315 Zinc-Finger Nucleases 315
T-Cells Chemical Conjugates 225 Immunotherapy 225 Lentivirus Vectors 225
Targeted Gene Therapy Nanobiotechnology 339 Non-Viral Gene Delivery 339 Ophthalmic Diseases 339 Targeting AAV Vectors Chimeric Antigen Receptors
251 251
T-Cell Development, Infections, Notch, Thymus Cell Processing 425 Genetic Diseases 425 Immunotherapy 425
Molecular Therapy Volume 24, Supplement 1, May 2016 Copyright © The American Society of Gene & Cell Therapy
Telomerase Cancer - Cell Therapy 276 Hematologic Diseases 276 Immunotherapy 276 Thalassemia Crispr/Cas9 692 Hematologic Diseases 692 TALENs 692 Therapeutic Gene Modulation Animal Models Hsv Vectors Thermostability AAV Vectors QC/QA Assays Vector Characterization/Biology
656 656 34 34 34
Tissue Engineering AAV Vectors 524, 702 Cardiovascular Diseases 246 Cell Delivery 340 Cell Therapy - Hematopoietic 239 Cell Therapy - Mesenchymal 246, 524 Cell Therapy - Muscle 240 Cell Therapy - Other 340, 452 Commercialisation 452 Crispr/Cas9 318 Immunotherapy 239 Induced Pluripotent Stem Cells 318 Lentivirus Vectors 240, 502 Musculo-Skeletal Disorders 502 Non-Viral Gene Delivery 491 Physcial Delviery 491 Vaccines 491 Vector Immunology/Host Responses 702 Tlr9 Signaling AAV Vectors Metabolic Diseases Vector Immunology/Host Responses
696 696 696
Toxicology Studies AAV Vectors 365, 468, 470 Cancer - Gene Therapy 475 Chimeric Antigen Receptors 399 Clinical Gene Therapy 470 Genetic Diseases 32 Hematopoietic Stem Cells 32 Immunotherapy 399 Lentivirus Vectors 33, 475 Metabolic Diseases 468 Musculo-Skeletal Disorders 386, 625 S347
KEYWORD INDEX Ophthalmic Diseases 33 Pharmacology/Toxicology 365 RNAi and shRNA 386, 625 Transgene Engineering AAV Vectors 456 Gene Expression 456 Hemophilia 456 Transposon Cell Therapy - ES Cells and iPS Cells Cell Therapy - Muscle Chimeric Antigen Receptors Non-Viral Gene Delivery
628 628 705 628, 705
Tumor Microenvironment Adenovirus Vectors 662 Cancer - Gene Therapy 662 Immunotherapy 662 Tumor-Specific Promoter Cancer - Gene Therapy Cancer - Targeted Therapy Hematologic Diseases
677 677 677
Tunneling Nanotubes Cell Delivery Cell Therapy - Hematopoietic Genetic Diseases
336 336 336
U1 Adaptors, iRGD, KRAS, MYC, Pancreatic Cancer Cancer - Targeted Therapy Oligonucleotide Based Therapies RNA Delivery Neurological Disorders Neuromuscular Disorders Oligonucleotide Based Therapies
262 262 262 259 259 259
Ultrasound Animal Models 492 Hemophilia 492 Non-Viral Gene Delivery 492 Urea Cycle Disorder AAV Vectors Cell Therapy - ES Cells and iPS Cells Gene Correction/Modification/Targeting Genetic Diseases Metabolic Diseases Neurological Disorders
45 347 347 45 347 45
Vaccines AAV Vectors 196, 722 Adenovirus Vectors 22, 36, 642 Cancer - Gene Therapy 643 Cancer - T-Cell Therapy 68, 191, 596 Cell Delivery 596 Clinical Gene Therapy 36 DNA Vaccines 436 DNA Viral Vectors 712 Immunotherapy 191, 196, 395, 642, 643 Infectious Diseases 22, 435, 440, 479 Musculo-Skeletal Disorders 722 Nanobiotechnology 600 S348
Non-Viral Gene Delivery Non-Viral Vectorology Oligonucleotide Based Therapies Oncolytic Virus Physcial Delviery Regulatory Issues Respiratory/Lung Disorder RNA Viral Vectors Tissue Engineering Vector Immunology/Host Responses Vector Production
435, 436, 491 479 440 68 491 465, 600 22 68 491 395 465, 712
Vector Characterization/Biology AAV Vectors 7, 8, 9, 10, 11, 12, 34, 82, 91, 218, 254, 255, 257, 293, 298, 301, 302, 304, 307, 389, 474, 478, 540, 541, 546, 547, 548, 607, 695, 727 Adenovirus Vectors 14, 17, 655 Animal Models 2 Cancer - Gene Therapy 409, 548 Cancer - Targeted Therapy 655 Cell Reprogramming 344 Clinical Gene Therapy 547 Crispr/Cas9 293 DNA Viral Vectors 14, 17, 107, 108, 254, 727 Epigenetic Regulation 8 Evolution and Development 389 Exosomes 594 Foamy Virus 1 Gene Expression 10, 11, 91, 289, 474, 546 Genetic Diseases 257 Hematologic Diseases 230, 473 Hematopoietic Stem Cells 1 Hsv Vectors 107 Humoral Immune Response 82 Insertional Mutagenesis 2 Lentivirus Vectors 2, 230, 457, 709 Metabolic Diseases 218, 355 Neurological Disorders 255, 301, 540 Neuromuscular Disorders 389 Non-Viral Gene Delivery 344, 355 Non-Viral Vectorology 289 Ophthalmic Diseases 594, 607 Precision Medicine 7 QC/QA Assays 34, 156, 473 Regulation 546 Respiratory/Lung Disorders 181 RNA Viral Vectors 1, 409 SERCaMP, ATV6, ER Stress 218 Single-Molecule, Real-Time Sequencing 7 Targeted Gene Expression 108, 181, 298, 307, 478, 695 Thermostability 34 Vector Immunology/Host Responses 82 Vector Production 9, 12, 156, 302, 304, 457, 709 Vector Transduction 594 Viral Evolution 541 Vector Immunology/Host Responses AAV Vectors 77, 78, 79, 80, 81, 82, 90, 185, 310, 378, 424, 467, 595, 696, 697, 698, 700, 701, 702 Adenovirus Vectors 517 Animal Models 63, 415, 437, 698 Antibody Response 77 Molecular Therapy Volume 24, Supplement 1, May 2016 Copyright © The American Society of Gene & Cell Therapy
KEYWORD INDEX Anti-Transgene 694 Cancer - Gene Therapy 403 Cancer - Targeted Therapy 208, 415, 517 CD8+ T Cells, Ovalbumin 79 Cell Therapy - Lung 438 Cell Therapy - Neuronal 208 Clinical Gene Therapy 185, 697 Exosomes 595 Gene Correction/Modification/Targeting 764 Gene Expression 310, 700, 701 Hemophilia 78 Hsv Vectors 518 Humoral Immune Response 82 Immunotherapy 51, 63, 81, 395, 518 Infectious Diseases 79, 437 Lentivirus Vectors 51, 764 Long Term Expression 424 Metabolic Diseases 696 Musculo-Skeletal Disorders 378 Neuromuscular Disorders 90 Ophthalmic Diseases 80, 694 Pre-Existing AAV Antibodies 698 QC/QA Assays 467 Reovirus 415 Retrovirus Vectors 403 RNA Viral Vectors 438 Tissue Engineering 702 Tlr9 Signaling 696 Vaccines 395 Vector Characterization/Biology 82 Vector Production AAV Vectors 9, 12, 87, 92, 94, 95, 97, 100, 101, 290, 292, 294, 302, 304, 308, 309, 460, 463, 471, 539, 549, 591, 672, 684, 710 Adenovirus Vectors 706, 713 Cancer - Gene Therapy 672, 703 Capsid Modification 95 Cell Processing 707 Cell Reprogramming 704 Cell Therapy - Hematopoietic 6 Chromatography 100 Clinical Gene Therapy 711 Continuous Flow Ultracentrifugation 97 Crude Lysate 290 Cystic Fibrosis 711 Database Development 591 Design Of Experiment 708 DNA Viral Vectors 87, 97, 294, 549, 712 Gen Expression 704 Gene Correction/Modification/Targeting 286 Gene Expression 292, 471 Gene Therapy 94 Genetic Diseases 101 Gmp Manufacturing 460, 710 Good Manufacturing Procedures 100, 308, 309, 460, 463, 591, 706, 710, 713 Hematopoietic Stem Cells 458 Hemophilia 92, 684 Hsv Vectors 94 Hybrid Vector 672 Infectious Diseases 539 Molecular Therapy Volume 24, Supplement 1, May 2016 Copyright © The American Society of Gene & Cell Therapy
Lentivirus Vectors
286, 288, 457, 458, 464, 703, 704, 707, 708, 709, 711 Orbital Shaken Bioreactors 463 Oversized Vector 92 QC/QA Assays 156, 290 Regulatory Issues 464, 465 RNA Viral Vectors 6, 288, 703 Vaccines 465, 712 Vector Characterization/Biology 9, 12, 156, 302, 304, 457, 709 Vector Purification 294 XLRP 101 Vector Purification AAV Vectors DNA Viral Vectors Vector Production
294 294 294
Vector Transduction Exosomes 594 Ophthalmic Diseases 594 Vector Characterization/Biology 594 Viral Evolution AAV Vectors Vector Characterization/Biology
541 541
Viral Transduction Enhancers Hematopoietic Stem Cells 536 Lentivirus Vectors 536 Nanobiotechnology 536 Wet Age-Related Macular Degeneration AAV Vectors Clinical Gene Therapy Ophthalmic Diseases
248 248 248
WT1 Cancer - T-Cell Therapy 193 Chimeric Antigen Receptors 193 Immunotherapy 193 XIST-Mediated Chromosome Silencing Gene Correction/Modification/Targeting Targeted Gene Expression Zinc-Finger Nucleases
315 315 315
XLRP AAV Vectors Genetic Diseases Vector Production
101 101 101
Zinc-Finger Nucleases AAV Vectors 133, 485 Adenovirus Vectors 484 Animal Models 38 Cancer - Cell Therapy 641 Cancer - Gene Therapy 752 Cancer - T-Cell Therapy 752 Cell Therapy - Hematopoietic 734 Gene Correction/Modification/Targeting 37, 38, 118, 127, 130, 133, 315, 485, 563, 566, 641, 765 Genetic Modification 734 Hematologic Diseases 118 S349
KEYWORD INDEX Hematopoietic Stem Cells 37, 734 Infectious Diseases 765 Lentivirus Vectors 127, 130, 566 Metabolic Diseases 484 Mucopolysaccharidosis 485 Multiple Myeloma 752 Nanobiotechnology 563 Targeted Gene Expression 315 XIST-Mediated Chromosome Silencing 315 β-Thalassemias, Hemoglobinopathies Hematopoietic Stem Cells Lentivirus Vectors
S350
222 222
Molecular Therapy Volume 24, Supplement 1, May 2016 Copyright © The American Society of Gene & Cell Therapy
