Annual

KEYWORD INDEX
AAV Vectors Gene Expression 10, 11, 60, 91, 98, 99, 158, 159, 164, 252,
AAV Vectors 724 270, 292, 299, 310, 370, 456, 471, 474, 545, 546, 608,
Adenovirus Vectors 102 610, 616, 618, 622, 700, 701, 726, 728
Age-Related Macular Degeneration 189 Gene Therapy 94
Aging 619 Genetic Diseases 27, 28, 45, 49, 57, 83, 101, 164, 165,
Alzheimer’s Disease 59 257, 265, 269, 300, 348, 350, 381, 481, 551, 606, 617,
Amyotrophic Lateral Sclerosis 606 621, 755
Animal Model 629 Glaucoma 188
Animal Models 50, 52, 54, 55, 56, 84, 96, 145, 168, 180, 186, GMP Manufacturing 460, 710
189, 223, 227, 256, 296, 337, 346, 350, 356, 361, 362, Good Manufacturing Procedures 100, 308, 309, 460, 463,
376, 377, 498, 503, 604, 612, 613, 615, 619, 698, 721, 591, 592, 710
725, 760 Hearing 269
Antibody Response 77 Hematologic Diseases 227, 760
Auditory Diseases 269 Hematopoietic Stem Cells 285, 316
Autoimmune Disease 84 Hemophilia 52, 78, 92, 223, 227, 253, 296, 456, 545, 550,
Axon Regeneration 271 684, 688, 760
Barcoding 542 High Quality Grade 592
Brain Delivery 618 HIV 44
Butyrylcholinesterase 60 HSV Vectors 94
Cancer - Cell Therapy 678 Human Immune Response to AAV 24
Cancer - Gene Therapy 215, 219, 323, 548, 669, 672 Humoral Immune Response 82
Cancer - Targeted Therapy 89, 219, 723 Hybrid Vector 672
Cancer - T-Cell Therapy 323 Immunotherapy 81, 196, 472, 555, 688, 699
Capsid Engineering 252 Induced Pluripotent Stem Cells 553, 556
Capsid Modification 95 Infectious Diseases 44, 79, 145, 539, 555, 699
Cardiovascular Diseases 89, 180, 297 Insulin Therapy 550
CCR5 43 Intrathecal Delivery 724
CD40L, X-HIGM, Gene Editing 316 Iron-Overload 220
CD8+ T Cells, Ovalbumin 79 Krabbe Disease 57
Cell Delivery 554 Lentiviral Vectors 322
Cell Reprogramming 266 Lentivirus Vectors 129, 305, 632, 728
Cell Therapy - Hematopoietic 123, 285 Liver 120
Cell Therapy - Mesenchymal 524, 554, 632 Long Term Expression 24, 50, 60, 305, 390, 424
Cell Therapy - Muscle 85 Luekocyte Adhesion Deficiency 285
Cell Therapy - Neuronal 99, 678 Lysosomal Storage Disease 348
Chimeric Antigen Receptors 251 Lysosomal Storage Diseases 361
Chromatography 100 Metabolic Disease 356
Clinical Gene Therapy 24, 83, 185, 248, 249, 297, 387, 470, Metabolic Diseases 48, 58, 86, 93, 157, 158, 159, 162, 168,
480, 497, 547, 592, 615, 697, 715, 755 218, 346, 349, 351, 352, 359, 366, 372, 382, 468, 611,
Continuous Flow Ultracentrifugation 97 696
CRISPR/Cas 569 Methylmalonic Acidemia 168
CRISPR/Cas9 39, 43, 119, 120, 266, 293, 303, 321, 322, 481, MicroRNA 220, 379, 606, 669, 737, 739
482, 543, 570 Minicircle 306
Crude Lysate 290 MPS1 93
Cytotoxic Transgene 215 Mucopolysaccharidosis 485
Database Development 591 Muscular Dystrophy 726
Delivery Techniques 29 Musculo-Skeletal Disorders 49, 322, 378, 379, 380, 381, 382,
Diabetes 50, 543 387, 480, 483, 497, 503, 552, 622, 624, 629, 630, 722,
DMD Dogs 721 726
DNA Viral Vectors 87, 97, 102, 253, 254, 294, 306, 311, 319, Natural History and Trial Development 715
549, 630, 727 Neuritin 371
Enteric Nervous System, Gastrointestinal Disorders 612 Neurological Disorders 27, 30, 45, 54, 55, 56, 57, 58, 59,
Epigenetic Regulation 8 157, 162, 186, 252, 255, 256, 271, 295, 301, 321, 337,
Evolution and Development 389 348, 349, 351, 352, 356, 361, 362, 366, 368, 370, 371,
Exon Skipping 624 372, 376, 540, 583, 604, 605, 610, 611, 612, 613, 616,
Exosomes 595 618, 619, 621, 724
Functional MRI (fMRI), Visual Pathways, Brain 265 Neuromuscular Disorders 85, 90, 377, 380, 389, 390, 498,
Gene Correction/Modification/Targeting 39, 43, 44, 48, 551, 552, 629, 715, 721
86, 93, 119, 120, 123, 129, 133, 165, 188, 295, 303, 311, Neurotrophic Factor, Parkinson 610
319, 359, 481, 482, 483, 485, 542, 543, 569, 570, 605, Novel Capsid 256
620, 725
Molecular Therapy Volume 24, Supplement 1, May 2016 S321

Copyright © The American Society of Gene & Cell Therapy
KEYWORD INDEX
Ophthalmic Diseases 28, 29, 31, 80, 83, 88, 96, 98, 188, 189, Adenovirus Cytokine
248, 249, 265, 268, 270, 306, 544, 553, 556, 607, 608, Cancer - Gene Therapy 405
617, 620, 739 Cancer - T-Cell Therapy 405
Optogenetic Therapy 270 Immunotherapy 405
Optogenetics 186
Orbital Shaken Bioreactors 463 Adenovirus Vectors
Osteoporosis, Alpha-1 Antitrypsin 632 AAV Vectors 102
Oversized Vector 92 Animal Models 16, 417, 519
Passive Immunization 699 Apoptosis 211, 659
Pharmacology/Toxicology 30, 31, 88, 268, 299, 365, 368, Breast Cancer 417
472, 550 Cancer - Cell Therapy 117, 420
Pitx3, Mitochondria, Antioxidants, Transcription 376 Cancer - Gene Therapy 16, 113, 117, 413, 414, 417, 418, 421,
Pompe Disease 49, 381 461, 509, 514, 516, 598, 659, 662
Precision Medicine 7 Cancer - Targeted Therapy 13, 103, 105, 211, 408, 421, 517,
Pre-Existing AAV Antibodies 698 519, 639, 655, 661
Promoterless Vector 48 Cancer - T-Cell Therapy 410, 513
Promoters 99 Cardiovascular Diseases 179
Prostate Cancer 669 Cell Delivery 420
QC/QA Assays 34, 290, 467 Cell Therapy - Hematopoietic 757
Re-Administration 472 Cell Therapy - Mesenchymal 453
Regulation 546 Checkpoint Inhibitor 513
Retina 544 Clinical Gene Therapy 36, 109, 110, 112, 115, 116, 453, 514,
RNAi and shRNA 145, 583, 737 661
Routes of Administration 300 CRISPR/Cas9 15, 104
SERCaMP, ATV6, ER Stress 218 DNA Viral Vectors 13, 14, 17, 102, 106
Single-Molecule, Real-Time Sequencing 7 Gene Correction/Modification/Targeting 15, 18
Somatic-Transgenic Technology 305 Gene Expression 109, 110, 112, 113, 115, 116, 324, 413
Spinal Muscular Atrophy, Type 1 480 Genetic Diseases 47
Sporadic Inclusion Body Myositis, Follistatin 497 Good Manufacturing Procedures 706, 713
TALENs 316, 569 Hematopoietic Stem Cells 106, 757
Targeted Gene Expression 29, 298, 300, 307, 478, 542, 695, Immunotherapy 408, 410, 414, 418, 509, 513, 516, 639, 642,
723 662
Targeting 251 Infectious Diseases 18, 22, 103
Thermostability 34 Keloid 112
Tissue Engineering 524, 702 Long Term Expression 324, 598
TLR9 Signaling 696 Mesothelioma 211, 661
Toxicology Studies 365, 468, 470 Metabolic Diseases 47, 179, 484
Transgene Engineering 456 Musculo-Skeletal Disorders 104
Urea Cycle Disorder 45 Novel Molecules to Enhance Viral Entry 176
Vaccines 196, 722 Pancreatic Cancer 639
Vector Characterization/Biology 7, 8, 9, 10, 11, 12, 34, Pharmacology/Toxicology 176
82, 91, 218, 254, 255, 257, 293, 298, 301, 302, 304, 307, QC/QA Assays 461
389, 474, 478, 540, 541, 546, 547, 548, 607, 695, 727 Respiratory/Lung Disorder 22
Vector Immunology/Host Responses 77, 78, 79, 80, 81, Respiratory/Lung Disorders 176
82, 90, 185, 310, 378, 424, 467, 595, 696, 697, 698, 700, RNAi and shRNA 105
701, 702 Tumor Microenvironment 662
Vector Production 9, 12, 87, 92, 94, 95, 97, 100, 101, 290, Vaccines 22, 36, 642
292, 294, 302, 304, 308, 309, 460, 463, 471, 539, 549, Vector Characterization/Biology 14, 17, 655
591, 672, 684, 710 Vector Immunology/Host Responses 517
Vector Purification 294 Vector Production 706, 713
Viral Evolution 541 Zinc-Finger Nucleases 484
Wet Age-Related Macular Degeneration 248
Adrenoleukodystrophy
XLRP 101
Clinical Gene Therapy 250
Zinc-Finger Nucleases 133, 485
Lentivirus Vectors 250
AAV Neurological Disorders 250
Animal Models 499
Age-Related Macular Degeneration
Gene Expression 499
AAV Vectors 189
Genetic Diseases 25
Animal Models 189
Metabolic Diseases 25
Ophthalmic Diseases 189
Musculo-Skeletal Disorders 499
Neurological Disorders 25
S322 Molecular Therapy Volume 24, Supplement 1, May 2016

KEYWORD INDEX
Aging HIV 427
AAV Vectors 619 HSV Vectors 656, 660, 667
Animal Models 619 Immunotherapy 63, 64, 275, 427, 693, 740, 749, 750
Neurological Disorders 619 Infectious Diseases 145, 173, 431, 437, 767
Injectable Biologic 26
Alzheimer’s Disease Insertional Mutagenesis 2
AAV Vectors 59 Lentivirus Vectors 2, 431, 631, 674, 756
CRISPR/Cas9 567 Liver Diseases 183
Gene Correction/Modification/Targeting 567 Long Term Expression 50, 693
Neurological Disorders 59, 567 Lysosomal Storage Deseases, Enzyme Replacement 430
Lysosomal Storage Diseases 361
Amyotrophic Lateral Sclerosis
Metabolic Disease 356
AAV Vectors 606
Metabolic Diseases 166, 168, 346, 357, 430, 477
Genetic Diseases 606
Methylmalonic Acidemia 168
MicroRNA 606
MicroRNA 529, 738
Animal Model Musculo-Skeletal Disorders 388, 499, 503, 633, 635
AAV Vectors 629 Neurological Disorders 26, 54, 55, 56, 186, 256, 337, 356,
Musculo-Skeletal Disorders 629 361, 362, 376, 604, 612, 613, 619, 627, 738
Neuromuscular Disorders 629 Neuromuscular Disorders 377, 388, 498, 623, 633, 721
Non-Invasive 633
Animal Models Non-Viral Gene Delivery 173, 490, 492
AAV 499 Notch Signaling 447
AAV Vectors 50, 52, 54, 55, 56, Novel Capsid 256
84, 96, 145, 168, 180, 186, 189, 223, 227, 256, 296, 337, Oligonucleotide Based Therapies 580, 623, 740
346, 350, 356, 361, 362, 376, 377, 498, 503, 604, 612, Oncolytic Virus 411
613, 615, 619, 698, 721, 725, 760 Ophthalmic Diseases 96, 189
Adenovirus Vectors 16, 417, 519 Optogenetics 186
Age-Related Macular Degeneration 189 Pitx3, Mitochondria, Antioxidants, Transcription 376
Aging 619 Pneumonia, Sepsis, Lung Contusion 173
Animal Models 84 Pre-Existing AAV Antibodies 698
Apoptosis 411 Reovirus 415
Autoimmune Disease 84 Respiratory/Lung Disorders 167, 182, 183, 490, 580
Bone Defect 631 Retrovirus Vectors 284
Breast Cancer 417 RNA Viral Vectors 205, 529, 673
Cancer - Gene Therapy 16, 205, 412, 417, 667, 673 RNAi and shRNA 145
Cancer - Targeted Therapy 411, 412, 415, 519, 660, 674 TALENs 758
Cancer - T-Cell Therapy 275 Targeted Gene Expression 26, 675
Cardiopulmonary 383 T-Cell Populations 693
Cardiovascular Diseases 180, 447, 448 Therapeutic Gene Modulation 656
Cell Therapy - Hematopoietic 427 Ultrasound 492
Cell Therapy - Lung 182 Vector Characterization/Biology 2
Cell Therapy - Mesenchymal 448 Vector Immunology/Host Responses 63, 415, 437, 698
Cell Therapy - Muscle 635 Zinc-Finger Nucleases 38
Cell Therapy - Other 430, 447
Chimeric Antigen Receptors 750 Anti CD19 CAR T KTE-C19
Clinical Gene Therapy 383, 615 Cancer - Cell Therapy 745
Colorectal Cancer 675 Cancer - T-Cell Therapy 745
CRISPR/Cas9 167 Chimeric Antigen Receptors 745
Cytokines 441
Antibody Delivery
Diabetes 50
Hematopoietic Stem Cells 429
DMD Dogs 721
Infectious Diseases 429
Enteric Nervous System, Gastrointestinal Disorders 612
Long Term Expression 429
Esophageal Squamous Cell Carcinoma 412
Ethical Issues 627 Antibody Response
Gastrointestinal Inflammation 477 AAV Vectors 77
Gene Correction/Modification/Targeting 38, 284, 725 Vector Immunology/Host Responses 77
Gene Expression 64, 441, 477, 499, 675, 749
Genetic Diseases 166, 183, 350, 357, 383, 631 Antisense Oligonucleotides
Hematologic Diseases 227, 760 Infectious Diseases 586
Hematopoietic Stem Cells 758, 767 Oligonucleotide Based Therapies 586
Hemophilia 52, 223, 227, 296, 492, 756, 760

KEYWORD INDEX
Anti-Transgene Brain Delivery
Ophthalmic Diseases 694 AAV Vectors 618
Vector Immunology/Host Responses 694 Gene Expression 618
Apoptosis
Adenovirus Vectors 211, 659 Breast Cancer
Animal Models 411 Adenovirus Vectors 417
Cancer - Gene Therapy 149, 345, 659 Animal Models 417
Cancer - Targeted Therapy 211, 411 Cancer - Gene Therapy 417
Chemical Conjugates 345
Curcumin, CDC, Breast Cancer 345 Butyrylcholinesterase
Mesothelioma 211 AAV Vectors 60
Oncolytic Virus 411 Gene Expression 60
RNA Delivery 149 Long Term Expression 60
Sendai Virus 149
Cancer - Cell Therapy
Aptamers AAV Vectors 678
Bioinformatics 148 Adenovirus Vectors 117, 420
Cancer - Targeted Therapy 671 Anti CD19 CAR T KTE-C19 745
Oligonucleotide Based Therapies 148, 744 Cancer - Gene Therapy 66, 117, 192, 207
Pharmacology/Toxicology 671 Cancer - Targeted Therapy 66, 204, 209, 216, 393
RNA Delivery 148, 744 Cancer - T-Cell Therapy 192, 199, 204, 393, 402, 515, 646,
652, 745, 748
Assay Development Cancer Stem Cells, Tumor Microenvironment 679
Clinical Gene Therapy 171 Cell Delivery 242, 420, 442, 511
Gene Expression 171 Cell Processing 402, 459
Hemophilia 171 Cell Reprogramming 645
Cell Therapy - ES Cells and iPS cells 528
Auditory Diseases Cell Therapy - Hematopoietic 233, 273
AAV Vectors 269 Cell Therapy - Mesenchymal 242
Genetic Diseases 269 Cell Therapy - Muscle 636
Hearing 269 Cell Therapy - Neuronal 678
Cell Therapy - Other 645
Autoimmune disease
Chimeric Antigen Receptor T Cells 204
AAV Vectors 84
Chimeric Antigen Receptors 207, 515, 745, 748
Animal Models 84
CRISPR/Cas9 422
Axon Regeneration Gene Correction/Modification/Targeting 641
AAV Vectors 271 Gene Expression 216, 679
Neurological Disorders 271 Good Manufacturing Procedures 459
Hematologic Diseases 233, 276
Barcoding Hematopoietic Stem Cells 273
AAV Vectors 542 Immunotherapy 199, 276, 422, 469, 476, 511, 646, 652
Gene Correction/Modification/Targeting 542 Integrase Defective Lentivirus Quantification 469
Targeted Gene Expression 542 Lentivirus Vectors 212, 462, 469, 476
Bioinformatics Nanobiotechnology 209, 442
Aptamers 148 Neurological Disorders 528
Clinical Gene Therapy 537 Oligonucleotide Based Therapies 263
Oligonucleotide Based Therapies 148 Oncolytic Adenovirus 515
Precision Medicine 537 Pharmacology/Toxicology 476
RNA Delivery 148 QC/QA Assays 263
RNA Viral Vectors 537 Reactive Oxygen Species 216
Respiratory/Lung Disorders 212
Bioprocess
RNA Viral Vectors 462
Cell Processing 343
Stem Cells; Ovarian Cancer; Drug Delivery 209
Cell Reprogramming 343
Targeted Gene Expression 679
Non-Viral Gene Delivery 343
Telomerase 276
Bone Defect Zinc-Finger Nucleases 641
Animal Models 631
Cancer - Gene Therapy
AAV Vectors 215, 219, 323, 548, 669, 672
Adenovirus Cytokine 405

KEYWORD INDEX
Adenovirus Vectors 16, 113, 117, 413, 414, 417, 418, 421, Vector Production 672, 703
461, 509, 514, 516, 598, 659, 662 Zinc-Finger Nucleases 752
Animal Models 16, 205, 412, 417, 667, 673
Apoptosis 149, 345, 659 Cancer - Targeted Therapy
Breast Cancer 417 AAV Vectors 89, 219, 723
Cancer - Cell Therapy 66, 117, 192, 207 Adenovirus Vectors 13, 103, 105, 211, 408, 421, 517, 519,
Cancer - Targeted Therapy 66, 155, 217, 219, 412, 416, 419, 639, 655, 661
421, 658, 670, 677, 762 Animal Models 411, 412, 415, 519, 660, 674
Cancer - T-Cell Therapy 74, 192, 206, 323, 405, 506, 653, Apoptosis 211, 411
746, 747, 752, 762 Aptamers 671
Cell Therapy - Hematopoietic 523 Cancer - Cell Therapy 66, 204, 209, 216, 393
Cell Therapy - Lung 585 Cancer - Gene Therapy 66, 155, 217, 219, 412, 416, 419, 421,
Chemical Conjugates 345, 602 658, 670, 677, 762
Chimeric Antigen Receptor, CAR 670 Cancer - Targeted Therapy 670
Chimeric Antigen Receptors 74, 207, 747 Cancer - T-Cell Therapy 202, 204, 392, 393, 396, 505, 762
Clinical Gene Therapy 61, 514 Cardiovascular Diseases 89
Colorectal Cancer 664 Cell Therapy - Neuronal 208, 443
Curcumin, CDC, Breast Cancer 345 Chimeric Antigen Receptor T Cells 204
Cytotoxic Transgene 215 Chimeric Antigen Receptor, CAR 670
DNA Viral Vectors 419, 520, 664 Chimeric Antigen Receptors 392
Esophageal Squamous Cell Carcinoma 412 Clinical Gene Therapy 72, 661
Ex-Vivo Expansion 523 DNA Vaccines, Electroporation 401
Gene Expression 113, 142, 413 DNA Viral Vectors 13, 419
Hematologic Diseases 677 Esophageal Squamous Cell Carcinoma 412
Hematopoietic Stem Cells 523, 668 Gene Expression 216
HSV Vectors 184, 520, 658, 664, 667, 680 Glioma 665
Hybrid Vector 672 Hematologic Diseases 65, 663, 677
Immunotherapy 61, 142, 201, 206, 261, 405, 414, 418, 495, HSV Vectors 658, 660, 665, 666
509, 516, 643, 644, 653, 662, 746 Immunotherapy 202, 401, 408, 423, 505, 639, 647, 663, 666
In Vivo T Cell Engineering 506 Infectious Diseases 103
Lentivirus Vectors 214, 217, 475, 703 Lentivirus Vectors 70, 217, 647, 674
Long Term Expression 598 Mesothelioma 211, 217, 661
Measles Virus, Glioblastoma 62 MicroRNA 67
Mesothelioma 217 Mitochondrial Biogenesis 65
MicroRNA 62, 144, 154, 587, 669 Nanobiotechnology 209, 396, 494
Multiple Myeloma 752 Neural Stem Cells, Pediatric Brain Tumors 443
Neurological Disorders 184 Neurological Disorders 665
Non-Small Cell Lung Cancer 585 Non-Viral Gene Delivery 72, 155, 494
Non-Viral Gene Delivery 152, 155, 493, 495, 593, 599, 644, Oligonucleotide Based Therapies 262, 590
680 Oncolytic Virus 411
Non-Viral Vectorology 599 Oncolytic Viruses 67, 423, 654, 666
Oligonucleotide Based Therapies 261 Pancreatic Cancer 639
Oncolytic Vectors 520 Pharmacology/Toxicology 671
PDGF, Angiogenesis 152 Polymeric, PBAE 494
Platelets 668 Reactive Oxygen Species 216
Polymers 493 Reovirus 415
Prostate Cancer 669 RNA Delivery 262
QC/QA Assays 461 RNA Viral Vectors 67, 416, 423, 654
Retroviral Replicating Vector 61 RNAi and shRNA 70, 105
Retrovirus Vectors 403, 602 Stem Cells; Ovarian Cancer; Drug Delivery 209
RNA Delivery 149, 593 Synthetic Gene Delivery Systems 590
RNA Viral Vectors 62, 201, 205, 409, 416, 506, 673, 703 Tandem Bispecific Chimeric Antigen Receptors 392
RNAi and shRNA 144, 152, 154, 585, 587, 588, 599 Targeted Gene Expression 723
Sendai Virus 149 Tumor-Specific Promoter 677
Synthetic Gene Delivery Systems 493, 588 U1 Adaptors, iRGD, KRAS, MYC, Pancreatic Cancer 262
Targeted Gene Expression 214, 668 Vector Characterization/Biology 655
Toxicology Studies 475 Vector Immunology/Host Responses 208, 415, 517
Tumor Microenvironment 662
Cancer - T-Cell Therapy
Tumor-Specific Promoter 677
AAV Vectors 323
Vaccines 643
Adenovirus Cytokine 405
Vector Characterization/Biology 409, 548
Adenovirus Vectors 410, 513
Vector Immunology/Host Responses 403
KEYWORD INDEX
Animal Models 275 Cancer Stem Cells, Tumor Microenvironment
Anti CD19 CAR T KTE-C19 745 Cancer - Cell Therapy 679
Cancer - Cell Therapy 192, 199, 204, 393, 402, 515, 646, 652, Gene Expression 679
745, 748 Targeted Gene Expression 679
Cancer - Gene Therapy 74, 192, 206, 323, 405, 506, 653, 746,
747, 752, 762 Capsid Engineering
Cancer - Targeted Therapy 202, 204, 392, 393, 396, 505, 762 AAV Vectors 252
CAR T Cells; Lymphoma; Manufacturing 287 Gene Expression 252
CD19, Sleeping Beauty 278 Neurological Disorders 252
Cell Delivery 596
Capsid Modification
Cell Processing 287, 402, 455, 650, 714
AAV Vectors 95
Cell Therapy - ES Cells and iPS Cells 236
Vector Production 95
Cell Therapy - Hematopoietic 236
Cell Therapy - Mesenchymal 466 CAR T Cells; Lymphoma; Manufacturing
Checkpoint Inhibitor 513 Cancer - T-Cell Therapy 287
Chimeric Antigen Receptor T Cells 204 Cell Processing 287
Chimeric Antigen Receptors 69, 73, 74, 190, 193, 195, 198, Immunotherapy 287
200, 203, 210, 247, 277, 392, 394, 397, 398, 400, 406,
451, 507, 510, 512, 515, 640, 649, 650, 651, 676, 745, Cardiopulmonary
747, 748, 751 Animal Models 383
Clinical Gene Therapy 247, 272 Clinical Gene Therapy 383
Clonality, T-Cell Repertoire 232 Genetic Diseases 383
CRISPR/Cas9 404, 572
DNA Viral Vectors 194 Cardiovascular Diseases
Gene Correction/Modification/Targeting 121, 391, 394, 398, AAV Vectors 89, 180, 297
572 Adenovirus Vectors 179
Genetic Diseases 232 Animal Models 180, 447, 448
Glycobiology 510 Cancer - Targeted Therapy 89
Good Manufacturing Procedures 75, 203, 466, 714 Cell Delivery 174, 178
Hematologic Diseases 71, 213, 400, 637 Cell Therapy - Endothelial 174
IL13Ra2, GBM, Solid Tumors 76 Cell Therapy - ES Cells and iPS Cells 244
IL-4 751 Cell Therapy - Mesenchymal 246, 448
Immunotherapy 71, 73, 75, 76, 190, 191, 193, 194, 195, 197, Cell Therapy - Other 174, 178, 447
198, 199, 202, 203, 206, 232, 272, 275, 277, 278, 287, Chemical Conjugates 177
391, 405, 406, 410, 451, 455, 505, 507, 510, 512, 513, Clinical Gene Therapy 297
637, 638, 640, 646, 648, 649, 651, 652, 653, 746, 751 CRISPR/Cas9 20
In Vivo T Cell Engineering 506 Familial Hypercholesterolaemia 19
Inflammatory Toxicity 197 Gene Expression 172, 177
Lentiviral Vectors 195 Genetic Diseases 20, 358
Lentivirus Vectors 121, 404, 638, 648 Long Term Expression 172
Leukemia and Lymphoma 69, 210 Metabolic Diseases 179, 358
Meganuclease, AAV vectors 394 MicroRNA 19
Meganucleases 200 MitoTimer 358
Multiple Myeloma 752 Non-Viral Gene Delivery 19
Mutated Neoantigens 391 Notch Signaling 447
Nanobiotechnology 396 QC/QA Assays 244
Non-Viral Gene Delivery 278 Tissue Engineering 246
Non-Viral Vectorology 213
CCR5
Oncolytic Adenovirus 515
AAV Vectors 43
Oncolytic Virus 68
CRISPR/Cas9 43
PD-1 572
Gene Correction/Modification/Targeting 43
Pediatric Cancers - Solid Tumors 649
Process Modeling; Monte Carlo Simulations 650 CD19, Sleeping Beauty
RNA Viral Vectors 68, 506 Cancer - T-Cell Therapy 278
Sleeping Beauty Transposition; Minicircle 73 Immunotherapy 278
TALENs 397, 676 Non-Viral Gene Delivery 278
Tandem Bispecific Chimeric Antigen Receptors 392
Targeted Gene Expression 76 CD40L, X-HIGM, Gene Editing
Vaccines 68, 191, 596 AAV Vectors 316
WT1 193 Hematopoietic Stem Cells 316
Zinc-Finger Nucleases 752 TALENs 316

KEYWORD INDEX
CD8+ T Cells, Ovalbumin Lentivirus Vectors 704
AAV Vectors 79 Musculo-Skeletal Disorders 525
Infectious Diseases 79 Non-Viral Gene Delivery 343, 344
Vector Immunology/Host Responses 79 Targeted Gene Expression 40
Vector Characterization/Biology 344
Cell Delivery Vector Production 704
AAV Vectors 554
Adenovirus Vectors 420 Cell Therapy - Endothelial
Cancer - Cell Therapy 242, 420, 442, 511 Cardiovascular Diseases 174
Cancer - T-Cell Therapy 596 Cell Delivery 174
Cardiovascular Diseases 174, 178 Cell Therapy - Other 174
Cell Therapy - Endothelial 174
Cell Therapy - Hematopoietic 336, 521, 603 Cell Therapy - ES Cells and iPS Cells
Cell Therapy - Lung 21 Cancer - Cell Therapy 528
Cell Therapy - Mesenchymal 242, 554 Cancer - T-Cell Therapy 236
Cell Therapy - Neuronal 521 Cardiovascular Diseases 244
Cell Therapy - Other 174, 178, 340 Cell Therapy - Hematopoietic 234, 236
CRISPR/Cas9 496, 603 Cell Therapy - Muscle 565, 628
Duchenne Muscular Dystrophy; Exon Skipping 584 Clinical Gene Therapy 527
Exosomes 496 CRISPR/Cas9 527, 730
Genetic Diseases 21, 336 Gene Correction/Modification/Targeting 122, 243, 312, 332,
Immunotherapy 511 347, 730
Nanobiotechnology 442 Genetic Diseases 522
Neuromuscular Disorders 584 Hematologic Diseases 444, 522
Oligonucleotide Based Therapies 584 Induced Pluripotent Stem Cells 241, 243, 444
Tissue Engineering 340 Lentivirus Vector 234
Tunneling Nanotubes 336 Lentivirus Vectors 241
Vaccines 596 Lentivirus, DMD 565
Cell Processing Metabolic Diseases 347
Bioprocess 343 Musculo-Skeletal Disorders 312
Cancer - Cell Therapy 402, 459 Neurological Disorders 528
Cancer - T-Cell Therapy 287, 402, 455, 650, 714 Neuromuscular Disorders 730
CAR T Cells; Lymphoma; Manufacturing 287 Non-Viral Gene Delivery 628
Cell Reprogramming 343 QC/QA Assays 244
Cell Therapy - Hematopoietic 526 Respiratory/Lung Disorders 122
Cell Therapy - Mesenchymal 245 TALENs 332, 565
Cell Therapy - Neuronal 375 Transposon 628
Chimeric Antigen Receptors 650 Urea Cycle Disorder 347
Good Manufacturing Procedures 459, 714 Cell Therapy - Hematopoietic
GPCR, TDP-43, Cannabinoids 375 AAV Vectors 123, 285
Hematopoietic Stem Cells 526 Adenovirus Vectors 757
Immunotherapy 287, 425, 455 Animal Models 427
Lentivirus Vectors 707 Cancer - Cell Therapy 233, 273
MicroRNA 245 Cancer - Gene Therapy 523
Non-Viral Gene Delivery 343 Cancer - T-Cell Therapy 236
Pharmacology/Toxicology 375 Cell Delivery 336, 521, 603
Process Modeling; Monte Carlo Simulations 650 Cell Processing 526
T-Cell Development, Infections, Notch, Thymus 425 Cell Therapy - ES Cells and iPS Cells 234, 236
Vector Production 707 Cell Therapy - Neuronal 521
Cell Reprogramming Clinical Gene Therapy 235
AAV Vectors 266 CRISPR/Cas9 603
Bioprocess 343 Ex-Vivo Expansion 523
Cancer - Cell Therapy 645 Foamy Virus 683
Cell Processing 343 Gene Correction/Modification/Targeting 123, 432, 562
Cell Therapy - Muscle 525 Gene Expression 766
Cell Therapy - Other 645 Genetic Diseases 163, 336
CRISPR/Cas9 266 Genetic Modification 734
Gen Expression 704 Hematologic Diseases 233, 683, 763

KEYWORD INDEX
Hematopoietic Stem Cells 235, 273, 283, 285, 432, 523, 526, Gene Expression 449
562, 689, 734, 757 Lentivirus Vectors 240
HIV 427, 763 Lentivirus, DMD 565
Immunotherapy 239, 427, 685 Musculo-Skeletal Disorders 238, 446, 449, 525, 577, 635, 636
Infectious Diseases 685 Neuromuscular Disorders 85
Lentivirus Vector 234 Non-Viral Gene Delivery 628
Lentivirus Vectors 283, 766 TALENs 565
Long Term Expression 234, 689 Tissue Engineering 240
Luekocyte Adhesion Deficiency 285 Transposon 628
Macrophages, HSC 766
Metabolic Diseases 163 Cell Therapy - Neuronal
Neurological Disorders 374 AAV Vectors 99, 678
RNA Viral Vectors 6, 374, 683 Cancer - Cell Therapy 678
TALEN 562 Cancer - Targeted Therapy 208, 443
Tissue Engineering 239 Cell Delivery 521
Tunneling Nanotubes 336 Cell Processing 375
Vector Production 6 Cell Therapy - Hematopoietic 521
Zinc-Finger Nucleases 734 Gene Expression 99
GPCR, TDP-43, Cannabinoids 375
Cell Therapy - Lung Neural Stem Cells, Pediatric Brain Tumors 443
Animal Models 182 Pharmacology/Toxicology 375
Cancer - Gene Therapy 585 Promoters 99
Cell Delivery 21 Vector Immunology/Host Responses 208
Genetic Diseases 21 Cell Therapy - Other
Non-Small Cell Lung Cancer 585 Animal Models 430, 447
Pulmonary Macrophage Transplantation 35 Cancer - Cell Therapy 645
Respiratory/Lung Disorders 35, 182 Cardiovascular Diseases 174, 178, 447
RNA Viral Vectors 438 Cell Delivery 174, 178, 340
RNAi and shRNA 585 Cell reprogramming 645
Vector Immunology/Host Responses 438 Cell Therapy - Endothelial 174
Cell Therapy - Mesenchymal Cell Therapy - Lung 35
AAV Vectors 524, 554, 632 Cell Therapy - Muscle 446
Adenovirus Vectors 453 Commercialisation 452
Animal Models 448 Commercialization 718
Cancer - Cell Therapy 242 CRISPR/Cas9 573
Cancer - T-Cell Therapy 466 Gene Correction/Modification/Targeting 573
Cardiovascular Diseases 246, 448 Gene Expression 454
Cell Delivery 242, 554 Genetic Diseases 161
Cell Processing 245 Good Manufacturing Procedures 445
Cell Therapy - Muscle 238 Hematologic Diseases 763
Clinical Gene Therapy 453 HIV 763
Diabetes 354 Immunotherapy 426
Gene Correction/Modification/Targeting 354 Infectious Diseases 426
Good Manufacturing Procedures 466 Long Term Expression 161
Lentivirus Vectors 632 Lysosomal Storage Deseases, Enzyme Replacement 430
Metabolic Diseases 354 Magnetic Targeting, MRI 338
MicroRNA 245 Metabolic Diseases 430
Musculo-Skeletal Disorders 238 MicroRNA 338
Osteoporosis, Alpha-1 Antitrypsin 632 Musculo-Skeletal Disorders 446, 454
Tissue Engineering 246, 524 Non-Viral Gene Delivery 338
Notch Signaling 447
Cell Therapy - Muscle Ophthalmic Diseases 445
AAV Vectors 85 Pulmonary Macrophage Transplantation 35
Animal Models 635 Regulatory Issues 718
Cancer - Cell Therapy 636 Respiratory/Lung Disorders 35
Cell Reprogramming 525 Tissue Engineering 340, 452
Cell Therapy - ES Cells and iPS Cells 565, 628
Cell Therapy - Mesenchymal 238 Cellular Clearance
Cell Therapy - Other 446 Gene Expression 187
CRISPR/Cas9 577 Ophthalmic Diseases 187

KEYWORD INDEX
CGD Stem Cell Gene Correction Process Modeling; Monte Carlo Simulations 650
CRISPR/Cas9 41 Regulatory T Cell 434
Gene Correction/Modification/Targeting 41 Sleeping Beauty Transposition; Minicircle 73
Non-Viral Gene Delivery 41 TALENs 397, 676
Tandem Bispecific Chimeric Antigen Receptors 392
Checkpoint Inhibitor Targeting 251
Adenovirus Vectors 513 Toxicology Studies 399
Cancer - T-Cell Therapy 513 Transposon 705
Immunotherapy 513 WT1 193
Chemical Conjugates Chromatography
Apoptosis 345 AAV Vectors 100
Cancer - Gene Therapy 345, 602 Good Manufacturing Procedures 100
Cardiovascular Diseases 177 Vector Production 100
Curcumin, CDC, Breast Cancer 345
Gene Expression 177 Chronic Pain
Immunotherapy 225 HSV Vectors 111
Lentivirus Vectors 225 Neurological Disorders 111
Nanobiotechnology 342 Targeted Gene Expression 111
Retrovirus Vectors 602 Clinical Gene Therapy
T-Cells 225 AAV Vectors 24, 83, 185, 248, 249, 297, 387, 470, 480, 497,
547, 592, 615, 697, 715, 755
Chimeric Antigen Receptor T Cells Adenovirus Vectors 36, 109, 110, 112, 115, 116, 453, 514, 661
Cancer - Cell Therapy 204 Adrenoleukodystrophy 250
Cancer - Targeted Therapy 204 Animal Models 383, 615
Cancer - T-Cell Therapy 204 Assay Development 171
Bioinformatics 537
Chimeric Antigen Receptor, CAR Cancer - Gene Therapy 61, 514
Cancer - Gene Therapy 670 Cancer - Targeted Therapy 72, 661
Cancer - Targeted Therapy 670 Cancer - T-Cell Therapy 247, 272
Cardiopulmonary 383
Chimeric Antigen Receptors
Cardiovascular Diseases 297
AAV Vectors 251
Animal Models 750
Anti CD19 CAR T KTE-C19 745
Cell Therapy - Mesenchymal 453
Cancer - Cell Therapy 207, 515, 745, 748
Chimeric Antigen Receptors 247
Cancer - Gene Therapy 74, 207, 747
CRISPR/Cas9 326, 527, 731
Cancer - Targeted Therapy 392
Cystic Fibrosis 711
Cancer - T-Cell Therapy 69, 73, 74, 190, 193, 195, 198, 200,
Gene Correction/Modification/Targeting 280, 326, 731
203, 210, 247, 277, 392, 394, 397, 398, 400, 406, 451,
Gene Expression 109, 110, 112, 115, 116, 171
507, 510, 512, 515, 640, 649, 650, 651, 676, 745, 747,
Genetic Diseases 83, 224, 353, 383, 534, 755
748, 751
Good Manufacturing Procedures 592
Cell Processing 650
HCV, AAV 258
CRISPR/Cas9 274
Hematopoietic Stem Cells 235, 314, 364, 754
Hemophilia 171
Glycobiology 510
High Quality Grade 592
Human Immune Response to AAV 24
Immunotherapy 61, 272
Hemophilia 434
IL-4 751
Integration Sites 531
Immunotherapy 73, 190, 193, 195, 198, 203, 274, 277, 399,
Keloid 112
406, 434, 451, 507, 510, 512, 640, 649, 651, 750, 751,
Lentivirus Vectors 250, 279, 364, 531, 534, 711, 754
761
Lentiviral Vectors 195
Mathematical Modeling 314
Mesothelioma 661
Leukemia and Lymphoma 69, 210
Meganuclease, AAV Vectors 394
MicroRNA 282
Meganucleases 200
Musculo-Skeletal Disorders 328, 387, 480, 497
Natural History and Trial Development 715
Oncolytic Adenovirus 515
Neurological Disorders 250, 267, 367
KEYWORD INDEX
Neuromuscular Disorders 715 CCR5 43
Non-Viral Gene Delivery 72 Cell Delivery 496, 603
Oligonucleotide Based Therapies 328 Cell Reprogramming 266
Ophthalmic Diseases 83, 248, 249, 267 Cell Therapy - ES Cells and iPS Cells 527, 730
Precision Medicine 537 Cell Therapy - Hematopoietic 603
Regulatory Issues 719 Cell Therapy - Muscle 577
Retroviral Replicating Vector 61 Cell Therapy - Other 573
Retrovirus Vectors 224, 367, 531 CGD Stem Cell Gene Correction 41
RNA Viral Vectors 537 Chimeric Antigen Receptors 274
RNAi and shRNA 258 Clinical Gene Therapy 326, 527, 731
Spinal Muscular Atrophy, Type 1 480 Diabetes 543
Sporadic Inclusion Body Myositis, Follistatin 497 Dystrophin 561
Targeted Gene Expression 282 Epigenetic Regulation 131, 385, 729
Toxicology Studies 470 Exosomes 313, 496
Vaccines 36 Gene Correction/Modification/Targeting 3, 15, 39,
Vector Characterization/Biology 547 41, 43, 119, 120, 124, 126, 128, 131, 134, 136, 137, 139,
Vector Immunology/Host Responses 185, 697 140, 303, 313, 317, 326, 329, 330, 334, 481, 482, 487,
Vector Production 711 488, 501, 504, 543, 559, 561, 567, 568, 570, 571, 572,
Wet Age-Related Macular Degeneration 248 573, 575, 576, 729, 730, 731, 732, 733
Gene Expression 334
Clonality, T-Cell Repertoire Genetic Diseases 20, 481, 488, 561, 568, 576, 733
Cancer - T-Cell Therapy 232 Genomic Deletions 135
Genetic Diseases 232 Hematologic Diseases 140, 559, 571, 692
Immunotherapy 232 Hematopoietic Stem Cells 135, 330, 564, 732, 768
Hemophilia 128
Colorectal Cancer
HIV Latency 125
Animal Models 675
HIV-1, Virual Escape 331
Cancer - Gene Therapy 664
Homology-Directed Repair 568
DNA Viral Vectors 664
HSC Engraftment, Hematologic Disease 732
Gene Expression 675
Immunotherapy 274, 422, 768
HSV Vectors 664
Induced Pluripotent Stem Cells 318, 557, 626, 735
Infectious Diseases 125, 331, 574, 768
Commercialisation Lentiviral Vectors 322
Cell Therapy - Other 452 Lentivirus Vectors 3, 126, 134, 135, 327, 331, 404, 532, 564,
Tissue Engineering 452 574
Liver 120
Commercialization Musculo-Skeletal Disorders 104, 139, 317, 322, 385, 501,
Cell Therapy - Other 718 504, 577, 735
Regulatory Issues 718 Neurological Disorders 321, 327, 567
Neuromuscular Disorders 730
Common Rule Non-Viral Gene Delivery 41, 335, 487
Ethical Issues 717 Ophthalmic Diseases 124, 329
Regulatory Issues 717 PD-1 572
Precision Medicine 141, 575
Continuous Flow Ultracentrifugation
QC/QA Assays 141
AAV Vectors 97
Respiratory/Lung Disorders 23, 167
RNA Delivery 335
RNAi and shRNA 23, 532
CRISPR/Cas Synthetic Biology 136
AAV Vectors 569 TALENs 136, 137, 139, 692
Gene Correction/Modification/Targeting 569 Targeted Epigenetic Editing 729
TALENs 569 Targeted Gene Expression 125, 557, 626
Thalassemia 692
CRISPR/Cas9 Tissue Engineering 318
AAV Vectors 39, 43, 119, 120, 266, 293, 303, 321, 322, 481, Vector Characterization/Biology 293
482, 543, 570
Adenovirus Vectors 15, 104 Crude Lysate
Alzheimer’s Disease 567 AAV Vectors 290
Animal Models 167 QC/QA Assays 290
Cancer - Cell Therapy 422 Vector Production 290
Cancer - T-Cell Therapy 404, 572
KEYWORD INDEX
Curcumin, CDC, Breast Cancer Hematopoietic Stem Cells 106
Apoptosis 345 Hemophilia 253
Cancer - Gene Therapy 345 HSV Vectors 107, 520, 664
Chemical Conjugates 345 Immunotherapy 194
Minicircle 306
Cystic Fibrosis Musculo-Skeletal Disorders 630
Clinical Gene Therapy 711 Oncolytic Vectors 520
Lentivirus Vectors 711 Ophthalmic Diseases 306
Vector Production 711 Targeted Gene Expression 108
Vaccines 712
Cytokines
Vector Characterization/Biology 14, 17, 107, 108, 254, 727
Animal Models 441
Vector Production 87, 97, 294, 549, 712
Gene Expression 441
Vector Purification 294
Cytotoxic Transgene
Down Syndrome
AAV Vectors 215
Genetic Diseases 53
Database Development RNAi and shRNA 53
AAV Vectors 591
Duchenne Muscular Dystrophy; Exon Skipping
Cell Delivery 584
Delivery Techniques Oligonucleotide Based Therapies 584
AAV Vectors 29
Dystrophin
CRISPR/Cas9 561
Design of Experiment Genetic Diseases 561
Enteric Nervous System, Gastrointestinal Disorders
AAV Vectors 612
Diabetes Animal Models 612
AAV Vectors 50, 543 Neurological Disorders 612
Animal Models 50
Epigenetic Regulation
AAV Vectors 8
CRISPR/Cas9 543
CRISPR/Cas9 131, 385, 729
Gene Correction/Modification/Targeting 354, 543
Targeted Epigenetic Editing 729
DMD Dogs Vector Characterization/Biology 8
AAV Vectors 721
Esophageal Squamous Cell Carcinoma
Animal Models 721
Animal Models 412
DNA Vaccines, Electroporation Cancer - Targeted Therapy 412
Ethical Issues
Immunotherapy 401
Animal Models 627
DNA Vaccines Common Rule 717
Non-Viral Gene Delivery 436 Ethical Issues 717
Vaccines 436 Gene Correction/Modification/Targeting 720
Nanobiotechnology 720
DNA Viral Vectors Neurological Disorders 627
AAV Vectors 87, 97, 102, 253, 254, 294, 306, 311, 319, 549, Regulatory Issues 717
630, 727
Adenovirus Vectors 13, 14, 17, 102, 106 Evolution and Development
Cancer - Gene Therapy 419, 520, 664 AAV Vectors 389
Cancer - Targeted Therapy 13, 419 Neuromuscular Disorders 389
Cancer - T-Cell Therapy 194 Vector Characterization/Biology 389
Colorectal Cancer 664
Exon Skipping
Continuous Flow Ultracentrifugation 97
AAV Vectors 624
KEYWORD INDEX
Exosomes CCR5 43
AAV Vectors 595 Cell Reprogramming 40
Cell Delivery 496 Cell Therapy - ES Cells and iPS Cells 122, 243, 312, 332,
CRISPR/Cas9 313, 496 347, 730
Gene Correction/Modification/Targeting 313 Cell Therapy - Hematopoietic 123, 432, 562
Non-Viral Gene Delivery 341 Cell Therapy - Mesenchymal 354
Ophthalmic Diseases 594 Cell Therapy - Other 573
RNA Delivery 341 CGD Stem Cell Gene Correction 41
Vector Characterization/Biology 594 Chimeric Antigen Receptors 394, 398, 761
Vector Immunology/Host Responses 595 Clinical Gene Therapy 280, 326, 731
Vector Transduction 594 CRISPR/Cas 569
CRISPR/Cas9 3, 15, 39, 41, 43, 119, 120, 124, 126, 128,
Ex-Vivo Expansion 131, 134, 136, 137, 139, 140, 303, 313, 317, 326, 329,
Cancer - Gene Therapy 523 330, 334, 481, 482, 487, 488, 501, 504, 543, 559, 561,
Cell Therapy - Hematopoietic 523 567, 568, 570, 571, 572, 573, 575, 576, 729, 730, 731,
Hematopoietic Stem Cells 523 732, 733
Diabetes 354, 543
Familial Hypercholesterolaemia
DNA Viral Vectors 311, 319
Dystrophin 561
MicroRNA 19
Epigenetic Regulation 131, 729
Ethical Issues 720
Fanconi Anemia Exosomes 313
Gene Correction/Modification/Targeting 753 Fanconi Anemia 753
Genetic Diseases 753 Gene Expression 334, 560
Hematopoietic Stem Cells 753 Genetic Diseases 165, 226, 325, 481, 488, 558, 561, 568, 576,
579, 601, 733, 753
Foamy Virus Glaucoma 188
Cell Therapy - Hematopoietic 683 Hematologic Diseases 42, 118, 140, 280, 559, 560, 571, 682
Hematologic Diseases 683 Hematopoietic Stem Cells 37, 42, 330, 432, 558, 562, 732,
Hematopoietic Stem Cells 1 753
RNA Viral Vectors 1, 683 Hemophilia 128
Vector Characterization/Biology 1 HIV 44
Homology-Directed Repair 568
FSHD HSC Engraftment, Hematologic Disease 732
Musculo-Skeletal Disorders 153 Immunodeficiency 325
Oligonucleotide Based Therapies 153 Immunotherapy 132, 391, 761
Targeted Gene Expression 153 Induced Pluripotent Stem Cells 243
Infectious Diseases 18, 44, 765
Functional MRI (fMRI), Visual Pathways, Brain
Inflammation 226
AAV Vectors 265
Lentivirus Vectors 3, 121, 126, 127, 129, 130, 134, 226, 286,
533, 538, 566, 682, 764
Liver 120
Gastrointestinal Inflammation Meganuclease, AAV Vectors 394
Animal Models 477 Meganucleases 138, 579
Gene Expression 477 Metabolic Diseases 48, 86, 93, 347, 354, 359
Metabolic Diseases 477 MicroRNA 742
MPS1 93
Gen Expression Mucopolysaccharidosis 485
Cell Reprogramming 704 Musculo-Skeletal Disorders 139, 312, 317, 483, 501, 504
Lentivirus Vectors 704 Mutated Neoantigens 391
Vector Production 704 Nanobiotechnology 563, 720
Neurological Disorders 295, 567, 605, 742
Gene Correction/Modification/Targeting Neuromuscular Disorders 138, 730
AAV Vectors 39, 43, 44, 48, 86, 93, 119, 120, 123, 129, 133, Non-Viral Gene Delivery 41, 487, 601
165, 188, 295, 303, 311, 319, 359, 481, 482, 483, 485, Non-Viral Vectorology 533
542, 543, 569, 570, 605, 620, 725 Ophthalmic Diseases 124, 188, 329, 620
Adenovirus Vectors 15, 18 PD-1 572
Alzheimer’s Disease 567 Precision Medicine 575
Animal Models 38, 284, 725 Promoterless Vector 48
Barcoding 542 Respiratory/Lung Disorders 122
Cancer - Cell Therapy 641 Retrovirus Vectors 284
Cancer - T-Cell Therapy 121, 391, 394, 398, 572 RNA Delivery 538
KEYWORD INDEX
Synthetic Biology 136 Pharmacology/Toxicology 299
TALEN 562 Promoters 99
TALENs 132, 136, 137, 139, 325, 332, 569 Reactive Oxygen Species 216
Targeted Epigenetic Editing 729 Regulation 546
Targeted Gene Expression 40, 315, 542 Respiratory/Lung Disorders 743
Urea Cycle Disorder 347 RNA Viral Vectors 681
Vector Immunology/Host Responses 764 Targeted Gene Expression 320, 675, 679
Vector Production 286 Transgene Engineering 456
XIST-Mediated Chromosome Silencing 315 Vector Characterization/Biology 10, 11, 91, 289, 474, 546
Zinc-Finger Nucleases 37, 38, 118, 127, 130, 133, 315, 485, Vector Immunology/Host Responses 310, 700, 701
563, 566, 641, 765 Vector Production 292, 471
Gene Expression Gene Therapy

AAV 499 AAV Vectors 94
AAV Vectors 10, 11, 60, 91, 98, 99, 158, 159, 164, 252, 270, HSV Vectors 94
292, 299, 310, 370, 456, 471, 474, 545, 546, 608, 610, Vector Production 94
616, 618, 622, 700, 701, 726, 728
Adenovirus Vectors 109, 110, 112, 113, 115, 116, 324, 413 Genetic Diseases
Animal Models 64, 441, 477, 499, 675, 749 AAV 25
Assay Development 171 AAV Vectors 27, 28, 45, 49, 57, 83, 101, 164, 165, 257, 265,
brain Delivery 618 269, 300, 348, 350, 381, 481, 551, 606, 617, 621, 755
Butyrylcholinesterase 60 Adenovirus Vectors 47
Cancer - Cell Therapy 216, 679 Amyotrophic Lateral Sclerosis 606
Cancer - Gene Therapy 113, 142, 413 Animal Models 166, 183, 350, 357, 383, 631
Cancer - Targeted Therapy 216 Auditory Diseases 269
Cancer Stem Cells, Tumor Microenvironment 679 Bone Defect 631
Capsid Engineering 252 Cancer - T-Cell Therapy 232
Cardiovascular Diseases 172, 177 Cardiopulmonary 383
Cell Therapy - Hematopoietic 766 Cardiovascular Diseases 20, 358
Cell Therapy - Muscle 449 Cell Delivery 21, 336
Cell Therapy - Neuronal 99 Cell Processing 425
Cell Therapy - Other 454 Cell Therapy - ES Cells and iPS Cells 522
Cellular Clearance 187 Cell Therapy - Hematopoietic 163, 336
Chemical Conjugates 177 Cell Therapy - Lung 21
Clinical Gene Therapy 109, 110, 112, 115, 116, 171 Cell Therapy - Other 161
Colorectal Cancer 675 Clinical Gene Therapy 83, 224, 353, 383, 534, 755
CRISPR/Cas9 334 Clonality, T-Cell Repertoire 232
Cytokines 441 CRISPR/Cas9 20, 481, 488, 561, 568, 576, 733
Gastrointestinal Inflammation 477 Down Syndrome 53
Gene Correction/Modification/Targeting 334, 560 Dystrophin 561
Genetic Diseases 164, 373 Fanconi Anemia 753
Hematologic Diseases 560 Functional MRI (fMRI), Visual Pathways, Brain 265
Hemophilia 5, 171, 456, 545 Gene Correction/Modification/Targeting 165, 226, 325, 481,
Immunotherapy 64, 142, 749 488, 558, 561, 568, 576, 579, 601, 733, 753
Infectious Diseases 681 Gene Expression 164, 373
Inflammation 146 GM2 Gangliosidosis 363
Insertional Mutagenesis 681 Hearing 269
Keloid 112 Hematologic Diseases 522, 581
Lentivirus Vectors 5, 609, 728, 766 Hematopoietic Stem Cells 32, 291, 558, 753
Long Term Expression 60, 172, 324, 609 Homology-Directed Repair 568
Macrophages, HSC 766 Immunodeficiency 325
Metabolic Diseases 158, 159, 477 Immunotherapy 232, 425
MicroRNA 146 Inflammation 226
Minicircle 743 Krabbe Disease 57
Muscular Dystrophy 726 Lentivirus Vectors 226, 291, 534, 631
Musculo-Skeletal Disorders 449, 454, 499, 622, 726 Liver Diseases 183
Neurological Disorders 252, 370, 373, 610, 616, 618, 736 Long Term Expression 161, 439
Neurotrophic Factor, Parkinson 610 Lysosomal Storage Disease 348
Non-Viral Vectorology 289, 743 Meganucleases 579
Oligonucleotide Based Therapies 146 Metabolic Diseases 25, 46, 47, 160, 163, 166, 169, 353, 357,
Ophthalmic Diseases 98, 187, 270, 320, 608, 736 358, 360, 369
Optogenetic Therapy 270 MicroRNA 606
KEYWORD INDEX
MitoTimer 358 Cell Therapy - Mesenchymal 466
Musculo-Skeletal Disorders 49, 381, 741 Cell Therapy - Other 445
Neurological Disorders 25, 27, 45, 53, 57, 160, 348, 360, 363, Chimeric Antigen Receptors 203
369, 373, 621, 741 Chromatography 100
Neuromuscular Disorders 150, 551 Clinical Gene Therapy 592
Non-Viral Gene Delivery 169, 439, 597, 601 Database Development 591
Oligonucleotide Based Therapies 150, 581, 741 GMP Manufacturing 460, 710
Ophthalmic Diseases 28, 46, 83, 265, 617 High Quality Grade 592
Pompe Disease 49, 381 Immunotherapy 75, 203
Respiratory/Lung Disorders 183, 597 Ophthalmic Diseases 445
Retrovirus Vectors 224 Orbital Shaken Bioreactors 463
RNAi and shRNA 53 Vector Production 100, 308, 309, 460, 463, 591, 706, 710, 713
Routes of Administration 300
Splice, X-Linked Agammaglobulinemia 581 GPCR, TDP-43, Cannabinoids
TALENs 325 Cell Processing 375
Targeted Gene Expression 300 Cell Therapy - Neuronal 375
T-Cell Development, Infections, Notch, Thymus 425 Pharmacology/Toxicology 375
Toxicology Studies 32
HBV, AAV
Tunneling Nanotubes 336
Urea Cycle Disorder 45
RNAi and shRNA 260
XLRP 101 HCV, AAV
Genetic Modification
MicroRNA 143
Regulatory Issues 143
Zinc-Finger Nucleases 734
RNAi and shRNA 143, 258
Genomic Deletions
Hearing
CRISPR/Cas9 135
AAV Vectors 269
Auditory Diseases 269
Glaucoma
Hematologic Diseases
AAV Vectors 188
AAV Vectors 227, 760
Animal Models 227, 760
Cancer - Cell Therapy 233, 276
Glioma Cancer - Gene Therapy 677
Cancer - Targeted Therapy 665 Cancer - Targeted Therapy 65, 663, 677
HSV Vectors 665 Cancer - T-Cell Therapy 71, 213, 400, 637
Neurological Disorders 665 Cell Therapy - ES Cells and iPS Cells 444, 522
Cell Therapy - Hematopoietic 233, 683, 763
Glycobiology Cell Therapy - Other 763
Cancer - T-Cell Therapy 510 Chimeric Antigen Receptors 400, 407
Chimeric Antigen Receptors 510 Clinical Gene Therapy 279, 280
Immunotherapy 510 CRISPR/Cas9 140, 559, 571, 692
Foamy Virus 683
GM2 Gangliosidosis Gene Correction/Modification/Targeting 42, 118, 140, 280,
Genetic Diseases 363 559, 560, 571, 682
Neurological Disorders 363 Gene Expression 560
Genetic Diseases 522, 581
GMP Manufacturing
Hematopoietic Stem Cells 4, 42, 221, 228, 229, 231, 281,
508, 759
Good Manufacturing Procedures 460, 710
Hemophilia 227, 760
Vector Production 460, 710
HIV 763
Good Manufacturing Procedures Immunotherapy 71, 276, 508, 637, 663
AAV Vectors 100, 308, 309, 460, 463, 591, 592, 710 Induced Pluripotent Stem Cells 444
Adenovirus Vectors 706, 713 Lentivirus Vectors 4, 221, 228, 229, 230, 279, 281, 407, 682,
Cancer - Cell Therapy 459 686, 690, 759
Cancer - T-Cell Therapy 75, 203, 466, 714 Mitochondrial Biogenesis 65
Cell Processing 459, 714 Mixed Chimerism 231

KEYWORD INDEX
Non-Viral Vectorology 213 Hemophilia
Oligonucleotide Based Therapies 581 AAV Vectors 52, 78, 92, 223, 227, 253, 296, 456, 545, 550,
Pharmacology/Toxicology 690 684, 688, 760
QC/QA Assays 473 Animal Models 52, 223, 227, 296, 492, 756, 760
RNA Viral Vectors 683 Assay Development 171
Splice, X-Linked Agammaglobulinemia 581 Chimeric Antigen Receptors 434
TALENs 686, 692 Clinical Gene Therapy 171
Telomerase 276 CRISPR/Cas9 128
Thalassemia 692 DNA Viral Vectors 253
Tumor-Specific Promoter 677 Gene Correction/Modification/Targeting 128
Vector Characterization/Biology 230, 473 Gene Expression 5, 171, 456, 545
Zinc-Finger Nucleases 118 Hematologic Diseases 227, 760
Hematopoietic Stem Cells Insulin Therapy 550
AAV Vectors 285, 316 Lentivirus Vectors 5, 756
Adenovirus Vectors 106, 757 Non-Viral Gene Delivery 492
Animal Models 758, 767 Oversized Vector 92
Antibody Delivery 429 Pharmacology/Toxicology 550
Cancer - Cell Therapy 273 Regulatory T Cell 434
Cancer - Gene Therapy 523, 668 Transgene Engineering 456
CD40L, X-HIGM, Gene Editing 316 Ultrasound 492
Cell Processing 526 Vector Immunology/Host Responses 78
Cell Therapy - Hematopoietic 235, 273, 283, 285, 432, 523, Vector Production 92, 684
526, 562, 689, 734, 757
Clinical Gene Therapy 235, 314, 364, 754 High Quality Grade
CRISPR/Cas9 135, 330, 564, 732, 768 AAV Vectors 592
DNA Viral Vectors 106 Clinical Gene Therapy 592
Ex-Vivo Expansion 523 Good Manufacturing Procedures 592
Fanconi Anemia 753
Foamy Virus 1 HIV Latency
Gene Correction/Modification/Targeting 37, 42, 330, 432, CRISPR/Cas9 125
558, 562, 732, 753 Infectious Diseases 125
Genetic Diseases 32, 291, 558, 753 Targeted Gene Expression 125
Genetic Modification 734
HIV Stem Cell Gene Therapy
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 HIV
Immunotherapy 508, 768 AAV Vectors 44
Infectious Diseases 333, 429, 687, 767, 768 Animal Models 427
Lentivirus Vectors 4, 135, 221, 222, 228, 229, 281, 283, 291, Cell Therapy - Hematopoietic 427, 763
333, 364, 458, 530, 536, 564, 754, 759 Cell Therapy - Other 763
Long Term Expression 429, 689 Gene Correction/Modification/Targeting 44
Luekocyte Adhesion Deficiency 285 Hematologic Diseases 763
Mathematical Modeling 314 Immunotherapy 427
Mixed Chimerism 231 Infectious Diseases 44
Platelets 668 HIV-1, Virual Escape
Retrovirus Vectors 530, 687 CRISPR/Cas9 331
RNA Viral Vectors 1 Infectious Diseases 331
TALEN 562 Lentivirus Vectors 331
TALENs 316, 758
Targeted Gene Expression 668 Homology-Directed Repair
Toxicology Studies 32 CRISPR/Cas9 568
Vector Characterization/Biology 1 Gene Correction/Modification/Targeting 568
Vector Production 458 Genetic Diseases 568
Viral Transduction Enhancers 536
HSC Engraftment, Hematologic Disease
Zinc-Finger Nucleases 37, 734
CRISPR/Cas9 732
β-Thalassemias, Hemoglobinopathies 222

KEYWORD INDEX
HSV Vectors Cancer - T-Cell Therapy 71, 73, 75, 76, 190, 191, 193, 194,
AAV Vectors 94 195, 197, 198, 199, 202, 203, 206, 232, 272, 275, 277,
Animal Models 656, 660, 667 278, 287, 391, 405, 406, 410, 451, 455, 505, 507, 510,
Cancer - Gene Therapy 184, 520, 658, 664, 667, 680 512, 513, 637, 638, 640, 646, 648, 649, 651, 652, 653,
Cancer - Targeted Therapy 658, 660, 665, 666 746, 751
Chronic Pain 111 CAR T Cells; Lymphoma; Manufacturing 287
Colorectal Cancer 664 CD19, Sleeping Beauty 278
DNA Viral Vectors 107, 520, 664 Cell Delivery 511
Gene Therapy 94 Cell Processing 287, 425, 455
Glioma 665 Cell Therapy - Hematopoietic 239, 427, 685
Immunotherapy 518, 666 Cell Therapy - Other 426
Neurological Disorders 111, 114, 184, 657, 665 Checkpoint Inhibitor 513
Non-Viral Gene Delivery 680 Chemical Conjugates 225
Oncolytic Vectors 520 Chimeric Antigen Receptors 73, 190, 193, 195, 198, 203, 274,
Oncolytic Viruses 666 277, 399, 406, 434, 451, 507, 510, 512, 640, 649, 651,
Retrovirus Vectors 657 750, 751, 761
Targeted Gene Expression 111, 114 Clinical Gene Therapy 61, 272
Therapeutic Gene Modulation 656 Clonality, T-Cell Repertoire 232
Vector Characterization/Biology 107 CRISPR/Cas9 274, 422, 768
Vector Immunology/Host Responses 518 DNA Vaccines, Electroporation 401
Vector Production 94 DNA Viral Vectors 194
Human Immune Response to AAV Gene Expression 64, 142, 749
AAV Vectors 24 Genetic Diseases 232, 425
Clinical Gene Therapy 24 Glycobiology 510
Long Term Expression 24 Good Manufacturing Procedures 75, 203
Hematologic Diseases 71, 276, 508, 637, 663
Humoral Immune Response
Hematopoietic Stem Cells 508, 768
AAV Vectors 82
Hemophilia 434, 688
HIV 427
HSV Vectors 518, 666
Hybrid Vector IL13Ra2, GBM, Solid Tumors 76
AAV Vectors 672 IL-4 751
Cancer - Gene Therapy 672 Infectious Diseases 264, 426, 428, 433, 555, 685, 699, 768
Vector Production 672 Inflammatory Toxicity 197
Integrase Defective Lentivirus Quantification 469
IL13Ra2, GBM, solid tumors Lentiviral Vectors 195
Cancer - T-Cell Therapy 76 Lentivirus Vectors 51, 225, 469, 476, 638, 647, 648
Immunotherapy 76 Long Term Expression 693
Targeted Gene Expression 76 Mutated Neoantigens 391
Non-Viral Gene Delivery 264, 278, 433, 495, 644
IL-4 Oligonucleotide Based Therapies 261, 740
Cancer - T-Cell Therapy 751 Oncolytic Viruses 423, 666
Chimeric Antigen Receptors 751 Pancreatic Cancer 639
Immunotherapy 751 Passive Immunization 699
Immunodeficiency
Pharmacology/Toxicology 472, 476
Re-Administration 472
Regulatory T Cell 434
TALENs 325
Retroviral Replicating Vector 61
Immunotherapy RNA Viral Vectors 201, 423
AAV Vectors 81, 196, 472, 555, 688, 699 Sleeping Beauty Transposition ; Minicircle 73
Adenovirus Cytokine 405 Synthetic Gene Delivery Systems 428
Adenovirus Vectors 408, 410, 414, 418, 509, 513, 516, 639, TALENs 132
642, 662 Targeted Gene Expression 76
Animal Models 63, 64, 275, 427, 693, 740, 749, 750 T-Cell Development, Infections, Notch, Thymus 425
Cancer - Cell Therapy 199, 276, 422, 469, 476, 511, 646, 652 T-Cell Populations 693
Cancer - Gene Therapy 61, 142, 201, 206, 261, 405, 414, 418, T-Cells 225
495, 509, 516, 643, 644, 653, 662, 746 Telomerase 276
Cancer - Targeted Therapy 202, 401, 408, 423, 505, 639, 647, Tissue Engineering 239
663, 666 Toxicology Studies 399
Tumor Microenvironment 662
KEYWORD INDEX
Vaccines 191, 196, 395, 642, 643 Inflammation
Vector Immunology/Host Responses 51, 63, 81, 395, 518 Gene Correction/Modification/Targeting 226
WT1 193 Gene Expression 146
In Vivo T Cell Engineering Lentivirus Vectors 226
Cancer - Gene Therapy 506 MicroRNA 146
Cancer - T-Cell Therapy 506 Oligonucleotide Based Therapies 146
Inflammatory Toxicity
Induced Pluripotent Stem Cells Cancer - T-Cell Therapy 197
AAV Vectors 553, 556 Immunotherapy 197
Cell Therapy - ES Cells and iPS Cells 241, 243, 444
CRISPR/Cas9 318, 557, 626, 735 Injectable Biologic
Gene Correction/Modification/Targeting 243 Animal Models 26
Hematologic Diseases 444 Neurological Disorders 26
Lentivirus Vectors 241 Targeted Gene Expression 26
Ophthalmic Diseases 553, 556 Insertional Mutagenesis
Targeted Gene Expression 557, 626 Animal Models 2
Tissue Engineering 318 Gene Expression 681
Infectious Diseases Lentivirus Vectors 2
AAV Vectors 44, 79, 145, 539, 555, 699 RNA Viral Vectors 681
Adenovirus Vectors 18, 22, 103 Vector Characterization/Biology 2
Animal Models 145, 173, 431, 437, 767
Antibody Delivery 429 Insulin Therapy
Antisense Oligonucleotides 586 AAV Vectors 550
Cancer - Targeted Therapy 103 Hemophilia 550
CD8+ T Cells, Ovalbumin 79 Pharmacology/Toxicology 550
Integrase Defective Lentivirus Quantification
Cancer - Cell Therapy 469
Immunotherapy 469
CRISPR/Cas9 125, 331, 574, 768
Gene Expression 681 Integration sites
HBV, AAV 260 Clinical Gene Therapy 531
HCV, AAV 258 Lentivirus Vectors 531
Hematopoietic Stem Cells 333, 429, 687, 767, 768 Retrovirus Vectors 531
HIV 44
HIV Latency 125 Intrathecal Delivery
HIV Stem Cell Gene Therapy 333 AAV Vectors 724
HIV-1, Virual Escape 331 Neurological Disorders 724
Immunotherapy 264, 426, 428, 433, 555, 685, 699, 768
Insertional Mutagenesis 681 Iron-Overload
Lentivirus Vectors 331, 333, 431, 574 AAV Vectors 220
Long Term Expression 260, 429 MicroRNA 220
Non-Viral Gene Delivery 173, 264, 433, 435
Keloid
Adenovirus Vectors 112
Oligonucleotide Based Therapies 440, 586
Passive Immunization 699
Gene Expression 112
Pneumonia, Sepsis, Lung Contusion 173
Respiratory/Lung Disorder 22 Krabbe Disease
Retrovirus Vectors 687 AAV Vectors 57
RNA Viral Vectors 681 Genetic Diseases 57
RNAi and shRNA 145, 258, 260 Neurological Disorders 57
Synthetic Gene Delivery Systems 428
Targeted Gene Expression 125 Lentiviral Vectors
Vaccines 22, 435, 440, 479 AAV Vectors 322
Vector Immunology/Host Responses 79, 437 Cancer - T-Cell Therapy 195
Vector Production 539 Chimeric Antigen Receptors 195
Zinc-Finger Nucleases 765 CRISPR/Cas9 322

KEYWORD INDEX
Immunotherapy 195 Retrovirus Vectors 530, 531
Musculo-Skeletal Disorders 322 RNA Delivery 538
RNA Viral Vectors 288, 462, 703
Lentivirus Vector RNAi and shRNA 70, 532, 589
Cell Therapy - ES Cells and iPS Cells 234 Somatic-Transgenic Technology 305
Cell Therapy - Hematopoietic 234 TALENs 686
Long Term Expression 234 Targeted Gene Expression 214
T-Cells 225
Lentivirus Vectors
Tissue Engineering 240, 502
AAV Vectors 129, 305, 632, 728
Toxicology studies 33, 475
Adrenoleukodystrophy 250
Vector Characterization/Biology 2, 230, 457, 709
Animal Models 2, 431, 631, 674, 756
Vector Immunology/Host Responses 51, 764
Bone Defect 631
Vector Production 286, 288, 457, 458, 464, 703, 704, 707,
Cancer - Cell Therapy 212, 462, 469, 476
708, 709, 711
Cancer - Gene Therapy 214, 217, 475, 703
Viral Transduction Enhancers 536
Cancer - Targeted Therapy 70, 217, 647, 674
Zinc-Finger Nucleases 127, 130, 566
Cancer - T-Cell Therapy 121, 404, 638, 648
β-Thalassemias, Hemoglobinopathies 222
Cell Processing 707
Cell Reprogramming 704 Lentivirus, DMD
Cell Therapy - ES Cells and iPS Cells 241 Cell Therapy - ES Cells and iPS Cells 565
Cell Therapy - Hematopoietic 283, 766 Cell Therapy - Muscle 565
Cell Therapy - Mesenchymal 632 TALENs 565
Cell Therapy - Muscle 240
Chemical Conjugates 225 Leukemia and Lymphoma
Chimeric Antigen Receptors 407 Cancer - T-Cell Therapy 69, 210
Clinical Gene Therapy 250, 279, 364, 531, 534, 711, 754 Chimeric Antigen Receptors 69, 210
CRISPR/Cas9 3, 126, 134, 135, 327, 331, 404, 532, 564, 574
Cystic Fibrosis 711 Liver Diseases
Design of Experiment 708 Animal Models 183
Gen Expression 704 Genetic Diseases 183
Gene Correction/Modification/Targeting 3, 121, 126, 127, Respiratory/Lung Disorders 183
129, 130, 134, 226, 286, 533, 538, 566, 682, 764
Liver
Gene Expression 5, 609, 728, 766
AAV Vectors 120
Genetic Diseases 226, 291, 534, 631
CRISPR/Cas9 120
Genomic Deletions 135
Hematologic Diseases 4, 221, 228, 229, 230, 279, 281, 407,
682, 686, 690, 759 Long Term Expression
Hematopoietic Stem Cells 4, 135, 221, 222, 228, 229, 281, AAV Vectors 24, 50, 60, 305, 390, 424
283, 291, 333, 364, 458, 530, 536, 564, 754, 759 Adenovirus Vectors 324, 598
Hemophilia 5, 756 Animal Models 50, 693
HIV Stem Cell Gene Therapy 333 Antibody Delivery 429
HIV-1, Virual Escape 331 Butyrylcholinesterase 60
Immunotherapy 51, 225, 469, 476, 638, 647, 648 Cancer - Gene Therapy 598
Induced Pluripotent Stem Cells 241 Cardiovascular Diseases 172
Infectious Diseases 331, 333, 431, 574 Cell Therapy - ES Cells and iPS Cells 234
Inflammation 226 Cell Therapy - Hematopoietic 234, 689
Insertional Mutagenesis 2 Cell Therapy - Other 161
Integrase Defective Lentivirus Quantification 469 Clinical Gene Therapy 24
Integration Sites 531 Diabetes 50
Long Term Expression 305, 609 Gene Expression 60, 172, 324, 609
Macrophages, HSC 766 Genetic Diseases 161, 439
Mesothelioma 217 HBV, AAV 260
MicroRNA 589 Hematopoietic Stem Cells 429, 689
Musculo-Skeletal Disorders 502 Human Immune Response to AAV 24
Nanobiotechnology 536 Immunotherapy 693
Neurological Disorders 250, 327 Infectious Diseases 260, 429
Non-Viral Vectorology 533 Lentivirus Vector 234
Ophthalmic Diseases 33 Lentivirus Vectors 305, 609
Osteoporosis, Alpha-1 Antitrypsin 632 Neuromuscular Disorders 390
Pharmacology/Toxicology 476, 690 Non-Viral Gene Delivery 439
Regulatory Issues 464 RNAi and shRNA 260
Respiratory/Lung Disorders 212 Somatic-Transgenic Technology 305

KEYWORD INDEX
T-Cell Populations 693 Clinical Gene Therapy 661
Vector Immunology/Host Responses 424 Lentivirus Vectors 217
Luekocyte Adhesion Deficiency Metabolic Disease

AAV Vectors 285 AAV Vectors 356
Cell Therapy - Hematopoietic 285 Animal Models 356
Hematopoietic Stem Cells 285 Neurological Disorders 356
Lung Cancer Metabolic Diseases

Nanobiotechnology 489 AAV 25
Non-Viral Gene Delivery 489 AAV Vectors 48, 58, 86, 93, 157, 158, 159, 162, 168, 218,
RNAi and shRNA 489 346, 349, 351, 352, 359, 366, 372, 382, 468, 611, 696
Adenovirus Vectors 47, 179, 484
Lysosomal Storage Deseases, Enzyme Replacement Animal Models 166, 168, 346, 357, 430, 477
Animal Models 430 Cardiovascular Diseases 179, 358
Cell Therapy - Other 430 Cell Therapy - ES Cells and iPS Cells 347
Metabolic Diseases 430 Cell Therapy - Hematopoietic 163
Lysosomal Storage Disease
AAV Vectors 348
Diabetes 354
Gastrointestinal Inflammation 477
Lysosomal Storage Diseases Gene Correction/Modification/Targeting 48, 86, 93, 347, 354,
AAV Vectors 361 359
Animal Models 361 Gene Expression 158, 159, 477
Neurological Disorders 361 Genetic Diseases 25, 46, 47, 160, 163, 166, 169, 353, 357,
358, 360, 369
Macrophages, HSC Lysosomal Storage Deseases, Enzyme Replacement 430
Cell Therapy - Hematopoietic 766 Methylmalonic Acidemia 168
Gene Expression 766 MitoTimer 358
Lentivirus Vectors 766 MPS1 93
mRNA 170
Magnetic Targeting, MRI Musculo-Skeletal Disorders 382
Cell Therapy - Other 338 Neurological Disorders 25, 58, 157, 160, 162, 349, 351, 352,
MicroRNA 338 360, 366, 369, 372, 611
Non-Viral Gene Delivery 338 Non-Viral Gene Delivery 169, 355
Mathematical Modeling
Promoterless Vector 48
RNA Delivery 170
SERCaMP, ATV6, ER Stress 218
Measles Virus, Glioblastoma Synthetic Gene Delivery Systems 170
Cancer - Gene Therapy 62 TLR9 Signaling 696
MicroRNA 62 Toxicology Studies 468
RNA Viral Vectors 62 Urea Cycle Disorder 347
Vector Characterization/Biology 218, 355
Meganuclease, AAV Vectors Vector Immunology/Host Responses 696
Cancer - T-Cell Therapy 394 Zinc-Finger Nucleases 484
Gene Correction/Modification/Targeting 394 Methylmalonic Acidemia
AAV Vectors 168
Meganucleases Animal Models 168
Cancer - T-Cell Therapy 200 Metabolic Diseases 168
Gene Correction/Modification/Targeting 138, 579 MicroRNA
Genetic Diseases 579 AAV Vectors 220, 379, 606, 669, 737, 739
Neuromuscular Disorders 138 Amyotrophic Lateral Sclerosis 606
Mesothelioma Cancer - Gene Therapy 62, 144, 154, 587, 669
Adenovirus Vectors 211, 661 Cancer - Targeted Therapy 67
Apoptosis 211 Cardiovascular Diseases 19
Cancer - Gene Therapy 217 Cell Processing 245
Cancer - Targeted Therapy 211, 217, 661 Cell Therapy - Mesenchymal 245

KEYWORD INDEX
Cell Therapy - Other 338 Muscular Dystrophy
Clinical Gene Therapy 282 AAV Vectors 726
Familial Hypercholesterolaemia 19 Gene Expression 726
Gene Correction/Modification/Targeting 742 Musculo-Skeletal Disorders 726
Gene Expression 146
Genetic Diseases 606 Musculo-Skeletal Disorders
HCV, AAV 143 AAV 499
Inflammation 146 AAV Vectors 49, 322, 378, 379, 380, 381, 382, 387, 480, 483,
Iron-Overload 220 497, 503, 552, 622, 624, 629, 630, 722, 726
Lentivirus Vectors 589 Adenovirus Vectors 104
Magnetic Targeting, MRI 338 Animal Model 629
Measles Virus, Glioblastoma 62 Animal Models 388, 499, 503, 633, 635
Musculo-Skeletal Disorders 379 Cancer - Cell Therapy 636
Neurological Disorders 738, 742 Cell Reprogramming 525
Non-Viral Gene Delivery 19, 338 Cell Therapy - ES Cells and iPS Cells 312
Oligonucleotide Based Therapies 146 Cell Therapy - Mesenchymal 238
Oncolytic Viruses 67 Cell Therapy - Muscle 238, 446, 449, 525, 577, 635, 636
Ophthalmic Diseases 739 Cell Therapy - Other 446, 454
Prostate Cancer 669 Clinical Gene Therapy 328, 387, 480, 497
Regulatory Issues 143 CRISPR/Cas9 104, 139, 317, 322, 385, 501, 504, 577, 735
RNA Viral Vectors 62, 67, 529 DNA Viral Vectors 630
RNAi and shRNA 143, 144, 154, 587, 589, 737 Epigenetic regulation 385
Targeted Gene Expression 282 Exon Skipping 624
FSHD 153
Minicircle Gene Correction/Modification/Targeting 139, 312, 317, 483,
AAV Vectors 306 501, 504
DNA Viral Vectors 306 Gene Expression 449, 454, 499, 622, 726
Gene Expression 743 Genetic Diseases 49, 381, 741
Non-Viral Vectorology 743 Induced Pluripotent Stem Cells 735
Ophthalmic Diseases 306 Lentiviral Vectors 322
Respiratory/Lung Disorders 743 Lentivirus Vectors 502
Mitochondrial biogenesis MicroRNA 379
Cancer - Targeted Therapy 65 Muscular Dystrophy 726
Hematologic Diseases 65 Neurological Disorders 741
Neuromuscular Disorders 380, 388, 500, 552, 629, 633
MitoTimer
Non-Invasive 633
Oligonucleotide Based Therapies 153, 328, 741
Pompe Disease 49, 381
RNAi and shRNA 386, 500, 625
Mixed Chimerism Spinal Muscular Atrophy, Type 1 480
Hematologic Diseases 231 Sporadic Inclusion Body Myositis, Follistatin 497
Hematopoietic Stem Cells 231 TALENs 139
MPS1 Tissue Engineering 502
AAV Vectors 93 Toxicology Studies 386, 625
Gene Correction/Modification/Targeting 93 Vaccines 722
Metabolic Diseases 93 Vector Immunology/Host Responses 378
mRNA Mutated Neoantigens

Metabolic Diseases 170 Cancer - T-Cell Therapy 391
RNA Delivery 170 Gene Correction/Modification/Targeting 391
Synthetic Gene Delivery Systems 170 Immunotherapy 391
Mucopolysaccharidosis Nanobiotechnology
AAV Vectors 485 Cancer - Cell Therapy 209, 442
Gene Correction/Modification/Targeting 485 Cancer - Targeted Therapy 209, 396, 494
Zinc-Finger Nucleases 485 Cancer - T-Cell Therapy 396
Cell Delivery 442
Multiple Myeloma Chemical Conjugates 342
Cancer - Gene Therapy 752 Ethical Issues 720
Cancer - T-Cell Therapy 752 Gene Correction/Modification/Targeting 563, 720
KEYWORD INDEX
Hematopoietic Stem Cells 536 Lentivirus Vectors 250, 327
Lentivirus Vectors 536 Lysosomal Storage Disease 348
Lung Cancer 489 Lysosomal Storage Diseases 361
Non-Viral Gene Delivery 339, 489, 494 Metabolic Disease 356
Ophthalmic Diseases 339, 342 Metabolic Diseases 25, 58, 157, 160, 162, 349, 351, 352, 360,
Polymeric, PBAE 494 366, 369, 372, 611
Regulatory Issues 600 MicroRNA 738, 742
RNAi and shRNA 489 Musculo-Skeletal Disorders 741
Stem Cells; Ovarian Cancer; Drug Delivery 209 Neuritin 371
Targeted Gene Therapy 339 Neuromuscular Disorders 259
Vaccines 600 Neurotrophic Factor, Parkinson 610
Viral Transduction Enhancers 536 Novel Capsid 256
Zinc-Finger Nucleases 563 Oligonucleotide Based Therapies 259, 741
Ophthalmic Diseases 267, 736
Natural History and Trial Development Optogenetics 186
AAV Vectors 715 Pharmacology/Toxicology 30, 368
Clinical Gene Therapy 715 Pitx3, Mitochondria, Antioxidants, Transcription 376
Neuromuscular Disorders 715 Retrovirus Vectors 367, 657
Neural Stem Cells, Pediatric Brain Tumors
RNAi and shRNA 53, 583, 614
Cell Therapy - Neuronal 443
Targeted Gene Expression 26, 111, 114
Neuritin U1 Adaptors, Parkinsons, Huntingtons 259
AAV Vectors 371 Urea Cycle Disorder 45
Neurological Disorders 371 Vector Characterization/Biology 255, 301, 540
Neurological Disorders Neuromuscular Disorders

AAV 25 AAV Vectors 85, 90, 377, 380, 389, 390, 498, 551, 552, 629,
AAV Vectors 27, 30, 45, 54, 55, 56, 57, 58, 59, 157, 162, 715, 721
186, 252, 255, 256, 271, 295, 301, 321, 337, 348, 349, Animal Model 629
351, 352, 356, 361, 362, 366, 368, 370, 371, 372, 376, Animal Models 377, 388, 498, 623, 633, 721
540, 583, 604, 605, 610, 611, 612, 613, 616, 618, 619, Cell Delivery 584
621, 724 Cell Therapy - ES Cells and iPS Cells 730
Adrenoleukodystrophy 250 Cell Therapy - Muscle 85
Aging 619 Clinical Gene Therapy 715
Alzheimer’s Disease 59, 567 CRISPR/Cas9 730
Animal Models 26, 54, 55, 56, 186, 256, 337, 356, 361, 362, DMD Dogs 721
376, 604, 612, 613, 619, 627, 738 Duchenne Muscular Dystrophy; Exon Skipping 584
Axon Regeneration 271 Evolution and Development 389
Brain Delivery 618 Gene Correction/Modification/Targeting 138, 730
Cancer - Cell Therapy 528 Genetic Diseases 150, 551
Cancer - Gene Therapy 184 Long Term Expression 390
Cancer - Targeted Therapy 665 Meganucleases 138
Capsid engineering 252 Musculo-Skeletal Disorders 380, 388, 500, 552, 629, 633
Cell Therapy - ES Cells and iPS Cells 528 Natural History and Trial Development 715
Cell Therapy - Hematopoietic 374 Neurological Disorders 259
Chronic Pain 111 Non-Invasive 633
Clinical Gene Therapy 250, 267, 367 Oligonucleotide Based Therapies 150, 259, 584, 623
CRISPR/Cas9 321, 327, 567 RNAi and shRNA 500
Down Syndrome 53 U1 Adaptors, Parkinsons, Huntingtons 259
Enteric Nervous System, Gastrointestinal Disorders 612 Vector Characterization/Biology 389
Ethical Issues 627 Vector Immunology/Host Responses 90
Gene Correction/Modification/Targeting 295, 567, 605, 742
Neurotrophic Factor, Parkinson
Gene Expression 252, 370, 373, 610, 616, 618, 736
AAV Vectors 610
Genetic Diseases 25, 27, 45, 53, 57, 160, 348, 360, 363, 369,
Gene Expression 610
373, 621, 741
Glioma 665
GM2 Gangliosidosis 363 Non-Invasive
HSV Vectors 111, 114, 184, 657, 665 Animal Models 633
Injectable Biologic 26 Musculo-Skeletal Disorders 633
Intrathecal Delivery 724 Neuromuscular Disorders 633
Krabbe Disease 57
KEYWORD INDEX
Non-Small Cell Lung Cancer Infectious Diseases 479
Cancer - Gene Therapy 585 Lentivirus Vectors 533
Cell Therapy - Lung 585 Minicircle 743
RNAi and shRNA 585 Non-Viral Gene Delivery 599
Non-Viral Gene Delivery RNAi and shRNA 599
Animal Models 173, 490, 492 Vaccines 479
Bioprocess 343 Vector Characterization/Biology 289
Cancer - Gene Therapy 152, 155, 493, 495, 593, 599, 644, 680
Cancer - Targeted Therapy 72, 155, 494 Notch Signaling
Cancer - T-Cell Therapy 278 Animal Models 447
Cardiovascular Diseases 19 Cardiovascular Diseases 447
Cd19, Sleeping Beauty 278 Cell Therapy - Other 447
Cell Processing 343
Cell Reprogramming 343, 344 Novel Capsid
Cell Therapy - ES Cells and iPS Cells 628 AAV Vectors 256
Cell Therapy - Muscle 628 Animal Models 256
Cell Therapy - Other 338 Neurological Disorders 256
CGD Stem Cell Gene Correction 41
Novel Molecules to Enhance Viral Entry
Pharmacology/Toxicology 176
CRISPR/Cas9 41, 335, 487
DNA Vaccines 436
Exosomes 341 Oligonucleotide Based Therapies
Familial Hypercholesterolaemia 19 Animal Models 580, 623, 740
Gene Correction/Modification/Targeting 41, 487, 601 Antisense Oligonucleotides 586
Genetic Diseases 169, 439, 597, 601 Aptamers 148, 744
Hemophilia 492 Bioinformatics 148
HSV Vectors 680 Cancer - Cell Therapy 263
Immunotherapy 264, 278, 433, 495, 644 Cancer - Gene Therapy 261
Infectious Diseases 173, 264, 433, 435 Cancer - Targeted Therapy 262, 590
Long Term Expression 439 Cell Delivery 584
Lung Cancer 489 Clinical Gene Therapy 328
Magnetic Targeting, MRI 338 Duchenne Muscular Dystrophy; Exon Skipping 584
Metabolic Diseases 169, 355 FSHD 153
MicroRNA 19, 338 Gene Expression 146
Nanobiotechnology 339, 489, 494 Genetic Diseases 150, 581, 741
Non-Viral Vectorology 599 Hematologic Diseases 581
Ophthalmic Diseases 339 Immunotherapy 261, 740
PDGF, Angiogenesis 152 Infectious Diseases 440, 586
Physcial Delviery 491 Inflammation 146
Pneumonia, Sepsis, Lung Contusion 173 MicroRNA 146
Polymeric, PBAE 494 Musculo-Skeletal Disorders 153, 328, 741
Polymers 493 Neurological Disorders 259, 741
Respiratory/Lung Disorders 490, 597 Neuromuscular Disorders 150, 259, 584, 623
RNA Delivery 335, 341, 593 QC/QA Assays 263
RNAi and shRNA 152, 489, 599 Respiratory/Lung Disorders 580
Synthetic Gene Delivery Systems 493 RNA Delivery 148, 262, 744
Targeted Gene Therapy 339 Splice, X-Linked Agammaglobulinemia 581
Tissue Engineering 491 Synthetic Gene Delivery Systems 590
Transposon 628, 705 Targeted Gene Expression 153
Ultrasound 492 U1 Adaptors, iRGD, KRAS, MYC, Pancreatic Cancer 262
Vaccines 435, 436, 491 U1 Adaptors, Parkinsons, Huntingtons 259
Vector Characterization/Biology 344, 355 Vaccines 440
Non-Viral Vectorology Oncolytic Adenovirus
Cancer - Gene Therapy 599 Cancer - Cell Therapy 515
Cancer - T-Cell Therapy 213 Cancer - T-Cell Therapy 515
Gene Correction/Modification/Targeting 533 Chimeric Antigen Receptors 515
Gene Expression 289, 743
Hematologic Diseases 213

KEYWORD INDEX
Oncolytic Vectors Optogenetic Therapy
Cancer - Gene Therapy 520 AAV Vectors 270
DNA Viral Vectors 520 Gene Expression 270
Hsv Vectors 520 Ophthalmic Diseases 270
Oncolytic Virus Optogenetics

Animal Models 411 AAV Vectors 186
Apoptosis 411 Animal Models 186
Cancer - Targeted Therapy 411 Neurological Disorders 186
Cancer - T-Cell Therapy 68
RNA Viral Vectors 68 Orbital Shaken Bioreactors
Vaccines 68 AAV Vectors 463
Oncolytic Viruses Vector Production 463
HSV Vectors 666 Osteoporosis, Alpha-1 Antitrypsin
Immunotherapy 423, 666 AAV Vectors 632
MicroRNA 67 Cell Therapy - Mesenchymal 632
RNA Viral Vectors 67, 423, 654 Lentivirus Vectors 632
Ophthalmic Diseases Oversized Vector

AAV Vectors 28, 29, 31, 80, 83, 88, 96, 98, 188, 189, 248, AAV Vectors 92
249, 265, 268, 270, 306, 544, 553, 556, 607, 608, 617, Hemophilia 92
620, 739 Vector Production 92
Age-Related Macular Degeneration 189
Pancreatic Cancer
Anti-Transgene 694
Immunotherapy 639
Cellular Clearance 187
Chemical Conjugates 342 Passive Immunization
Clinical Gene Therapy 83, 248, 249, 267 AAV Vectors 699
CRISPR/Cas9 124, 329 Immunotherapy 699
Delivery Techniques 29 Infectious Diseases 699
Exosomes 594 PD-1
Functional MRI (fMRI), Visual Pathways, Brain 265 Cancer - T-Cell Therapy 572
Gene Correction/Modification/Targeting 124, 188, 329, 620 CRISPR/Cas9 572
Gene Expression 98, 187, 270, 320, 608, 736 Gene Correction/Modification/Targeting 572
Genetic Diseases 28, 46, 83, 265, 617
Glaucoma 188 PDGF, Angiogenesis
Good Manufacturing Procedures 445 Cancer - Gene Therapy 152
Induced Pluripotent Stem Cells 553, 556 Non-Viral Gene Delivery 152
Lentivirus Vectors 33 RNAi and shRNA 152
Pediatric Cancers - Solid Tumors
MicroRNA 739
Minicircle 306
Nanobiotechnology 339, 342
Immunotherapy 649
Neurological Disorders 267, 736
Non-Viral Gene Delivery 339 Pharmacology/Toxicology
Optogenetic Therapy 270 AAV Vectors 30, 31, 88, 268, 299, 365, 368, 472, 550
Pharmacology/Toxicology 31, 88, 268 Adenovirus Vectors 176
Retina 544 Aptamers 671
Targeted Gene Expression 29, 320 Cancer - Cell Therapy 476
Targeted Gene Therapy 339 Cancer - Targeted Therapy 671
Toxicology Studies 33 Cell Processing 375
Vector Characterization/Biology 594, 607 Cell Therapy - Neuronal 375
Vector Immunology/Host Responses 80, 694 Gene Expression 299
Vector Transduction 594 GPCR, TDP-43, Cannabinoids 375
Wet Age-Related Macular Degeneration 248 Hematologic Diseases 690
Hemophilia 550
Insulin Therapy 550

KEYWORD INDEX
Lentivirus Vectors 476, 690 Promoterless Vector
Neurological Disorders 30, 368 AAV Vectors 48
Novel Molecules to Enhance Viral Entry 176 Gene Correction/Modification/Targeting 48
Ophthalmic Diseases 31, 88, 268 Metabolic Diseases 48
Re-Administration 472
Respiratory/Lung Disorders 176 Promoters
Toxicology Studies 365 AAV Vectors 99
Cell Therapy - Neuronal 99
Physcial Delviery Gene Expression 99
Tissue Engineering 491 Prostate Cancer
Vaccines 491 AAV Vectors 669
Pitx3, Mitochondria, Antioxidants, Transcription MicroRNA 669
AAV Vectors 376
Animal Models 376 Pulmonary Macrophage Transplantation
Neurological Disorders 376 Cell Therapy - Lung 35
Platelets Respiratory/Lung Disorders 35
Hematopoietic Stem Cells 668 QC/QA Assays
Targeted Gene Expression 668 AAV Vectors 34, 290, 467
Pneumonia, Sepsis, Lung Contusion Cancer - Cell Therapy 263
Animal Models 173 Cancer - Gene Therapy 461
Infectious Diseases 173 Cardiovascular Diseases 244
Non-Viral Gene Delivery 173 Cell Therapy - ES Cells and iPS Cells 244
CRISPR/Cas9 141
Polymeric, PBAE Crude lysate 290
Cancer - Targeted Therapy 494 Hematologic Diseases 473
Nanobiotechnology 494 Oligonucleotide Based Therapies 263
Non-Viral Gene Delivery 494 Precision Medicine 141
Thermostability 34
Polymers
Vector Characterization/Biology 34, 156, 473
Reactive Oxygen Species
Pompe Disease
AAV Vectors 49, 381
Gene Expression 216
Musculo-Skeletal Disorders 49, 381
Re-Administration
Precision Medicine
AAV Vectors 472
AAV Vectors 7
Immunotherapy 472
Bioinformatics 537
CRISPR/Cas9 141, 575 Regulation
Gene Correction/Modification/Targeting 575 AAV Vectors 546
QC/QA Assays 141 Gene Expression 546
RNA Viral Vectors 537 Vector Characterization/Biology 546
Single-Molecule, Real-Time Sequencing 7
Vector Characterization/Biology 7 Regulatory Issues
Pre-Existing AAV Antibodies Clinical Gene Therapy 719
AAV Vectors 698 Commercialization 718
Animal Models 698 Common Rule 717
Vector Immunology/Host Responses 698 Ethical Issues 717
HCV, AAV 143
Process Modeling; Monte Carlo Simulations
MicroRNA 143
Cell Processing 650
Regulatory Issues 719

KEYWORD INDEX
RNAi and shRNA 143 Lentivirus Vectors 530, 531
Vaccines 465, 600 Neurological Disorders 367, 657
Vector Production 464, 465 Vector Immunology/Host Responses 403
Regulatory T Cell RNA Delivery

Chimeric Antigen Receptors 434 Apoptosis 149
Hemophilia 434 Aptamers 148, 744
Immunotherapy 434 Bioinformatics 148
Cancer - Gene Therapy 149, 593
Reovirus Cancer - Targeted Therapy 262
Animal Models 415 CRISPR/Cas9 335
Cancer - Targeted Therapy 415 Exosomes 341
Vector Immunology/Host Responses 415 Gene Correction/Modification/Targeting 538
Respiratory/Lung Disorder
mRNA 170
Non-Viral Gene Delivery 335, 341, 593
Vaccines 22
Oligonucleotide Based Therapies 148, 262, 744
Respiratory/Lung Disorders Sendai Virus 149
Adenovirus Vectors 176 Synthetic Gene Delivery Systems 170
Animal Models 167, 182, 183, 490, 580 U1 Adaptors, iRGD, KRAS, MYC, Pancreatic Cancer 262
RNA Viral Vectors
Animal Models 205, 529, 673
Cell Therapy - Lung 35, 182
Bioinformatics 537
CRISPR/Cas9 23, 167
Cancer - Gene Therapy 62, 201, 205, 409, 416, 506, 673, 703
Gene Expression 743
Cancer - T-Cell Therapy 68, 506
Cell Therapy - Hematopoietic 6, 374, 683
Cell Therapy - Lung 438
Liver Diseases 183
Minicircle 743
Foamy Virus 1, 683
Non-Viral Gene Delivery 490, 597
Gene Expression 681
Novel Molecules to Enhance Viral Entry 176
Oligonucleotide Based Therapies 580
In Vivo T Cell Engineering 506
Pulmonary Macrophage Transplantation 35
RNAi and shRNA 23
Insertional Mutagenesis 681
Lentivirus Vectors 288, 462, 703
Measles Virus, Glioblastoma 62
Retina MicroRNA 62, 67, 529
AAV Vectors 544 Neurological Disorders 374
Ophthalmic Diseases 544 Oncolytic Virus 68
Oncolytic Viruses 67, 423, 654
Retroviral Replicating Vector Precision Medicine 537
Cancer - Gene Therapy 61 Vaccines 68
Clinical Gene Therapy 61 Vector Characterization/Biology 1, 409
Immunotherapy 61 Vector Immunology/Host Responses 438
Vector Production 6, 288, 703
Retrovirus Vectors
Animal Models 284 RNAi and shRNA
Cancer - Gene Therapy 403, 602 AAV Vectors 145, 583, 737
Chemical Conjugates 602 Adenovirus Vectors 105
Clinical Gene Therapy 224, 367, 531 Animal Models 145
Gene Correction/Modification/Targeting 284 Cancer - Gene Therapy 144, 152, 154, 585, 587, 588, 599
Genetic Diseases 224 Cancer - Targeted Therapy 70, 105
Hematopoietic Stem Cells 530, 687 Cell Therapy - Lung 585
Hsv Vectors 657 Clinical Gene Therapy 258
Infectious Diseases 687 Crispr/Cas9 23, 532
Integration Sites 531 Down Syndrome 53

KEYWORD INDEX
Genetic Diseases 53 Sporadic Inclusion Body Myositis, Follistatin
Hbv, AAV 260 AAV Vectors 497
Hcv, AAV 143, 258 Clinical Gene Therapy 497
Infectious Diseases 145, 258, 260 Musculo-Skeletal Disorders 497
Long Term Expression 260 Stem Cells; Ovarian Cancer; Drug Delivery
Lung Cancer 489 Cancer - Cell Therapy 209
MicroRNA 143, 144, 154, 587, 589, 737 Cancer - Targeted Therapy 209
Musculo-Skeletal Disorders 386, 500, 625 Nanobiotechnology 209
Synthetic Biology
CRISPR/Cas9 136
Non-Small Cell Lung Cancer 585
TALENs 136
Non-Viral Gene Delivery 152, 489, 599
Non-Viral Vectorology 599 Synthetic Gene Delivery Systems
Pdgf, Angiogenesis 152 Cancer - Gene Therapy 493, 588
Regulatory Issues 143 Cancer - Targeted Therapy 590
Respiratory/Lung Disorders 23 Immunotherapy 428
Synthetic Gene Delivery Systems 588, 614 Infectious Diseases 428
Toxicology Studies 386, 625 Metabolic Diseases 170
mRNA 170
Routes Of Administration
AAV Vectors 300
Polymers 493
Sendai Virus RNA Delivery 170
Apoptosis 149 RNAi and shRNA 588, 614
TALEN
RNA Delivery 149
SERCaMP, ATV6, ER Stress Gene Correction/Modification/Targeting 562
AAV Vectors 218 Hematopoietic Stem Cells 562
TALENs
Single-Molecule, Real-Time Sequencing Animal Models 758
AAV Vectors 7 Cancer - T-Cell Therapy 397, 676
Precision Medicine 7 CD40L, X-HIGM, Gene Editing 316
Vector Characterization/Biology 7 Cell Therapy - ES Cells and iPS Cells 332, 565
Sleeping Beauty Transposition ; Minicircle Chimeric Antigen Receptors 397, 676
Cancer - T-Cell Therapy 73 CRISPR/Cas 569
Chimeric Antigen Receptors 73 CRISPR/Cas9 136, 137, 139, 692
Immunotherapy 73 Gene Correction/Modification/Targeting 132, 136, 137, 139,
325, 332, 569
Somatic-Transgenic Technology Genetic Diseases 325
AAV Vectors 305 Hematologic Diseases 686, 692
Lentivirus Vectors 305 Hematopoietic Stem Cells 316, 758
Long Term Expression 305 Immunodeficiency 325
Immunotherapy 132
Spinal Muscular Atrophy, Type 1
AAV Vectors 480
Lentivirus, DMD 565
Synthetic Biology 136
Splice, X-Linked Agammaglobulinemia Thalassemia 692
Tandem Bispecific Chimeric Antigen Receptors

KEYWORD INDEX
Targeted Epigenetic Editing T-Cell Populations
CRISPR/Cas9 729 Animal Models 693
Epigenetic Regulation 729 Immunotherapy 693
Gene Correction/Modification/Targeting 729 Long Term Expression 693
Targeted Gene Expression T-Cells

AAV Vectors 29, 298, 300, 307, 478, 542, 695, 723 Chemical Conjugates 225
Animal Models 26, 675 Immunotherapy 225
Barcoding 542 Lentivirus Vectors 225
Cancer - Gene Therapy 214, 668 Telomerase
Cancer - Targeted Therapy 723 Cancer - Cell Therapy 276
Cancer - T-Cell Therapy 76 Hematologic Diseases 276
Cancer Stem Cells, Tumor Microenvironment 679 Immunotherapy 276
Cell Reprogramming 40
Thalassemia
Chronic Pain 111
Crispr/Cas9 692
Colorectal Cancer 675
TALENs 692
CRISPR/Cas9 125, 557, 626
Delivery Techniques 29 Therapeutic Gene Modulation
DNA Viral Vectors 108 Animal Models 656
FSHD 153 Hsv Vectors 656
Gene Expression 320, 675, 679 Thermostability
Genetic Diseases 300 AAV Vectors 34
Hematopoietic Stem Cells 668 QC/QA Assays 34
HIV Latency 125 Vector Characterization/Biology 34
HSV Vectors 111, 114
IL13Ra2, GBM, Solid Tumors 76 Tissue Engineering
Immunotherapy 76 AAV Vectors 524, 702
Induced Pluripotent Stem Cells 557, 626 Cardiovascular Diseases 246
Infectious Diseases 125 Cell Delivery 340
Injectable Biologic 26 Cell Therapy - Hematopoietic 239
Lentivirus Vectors 214 Cell Therapy - Mesenchymal 246, 524
MicroRNA 282 Cell Therapy - Muscle 240
Musculo-Skeletal Disorders 153 Cell Therapy - Other 340, 452
Neurological Disorders 26, 111, 114 Commercialisation 452
Oligonucleotide Based Therapies 153 Crispr/Cas9 318
Ophthalmic Diseases 29, 320 Immunotherapy 239
Platelets 668 Induced Pluripotent Stem Cells 318
Respiratory/Lung Disorders 181 Lentivirus Vectors 240, 502
Routes of Administration 300 Musculo-Skeletal Disorders 502
Vector Characterization/Biology 108, 181, 298, 307, 478, 695 Non-Viral Gene Delivery 491
Xist-Mediated Chromosome Silencing 315 Physcial Delviery 491
Zinc-Finger Nucleases 315 Vaccines 491
Targeted Gene Therapy
Nanobiotechnology 339 Tlr9 Signaling
Non-Viral Gene Delivery 339 AAV Vectors 696
Ophthalmic Diseases 339 Metabolic Diseases 696
Targeting
AAV Vectors 251 Toxicology Studies
Chimeric Antigen Receptors 251 AAV Vectors 365, 468, 470
T-Cell Development, Infections, Notch, Thymus Chimeric Antigen Receptors 399
Cell Processing 425 Clinical Gene Therapy 470
Genetic Diseases 425 Genetic Diseases 32
Immunotherapy 425 Hematopoietic Stem Cells 32
Immunotherapy 399
Lentivirus Vectors 33, 475
Musculo-Skeletal Disorders 386, 625
KEYWORD INDEX
Ophthalmic Diseases 33 Non-Viral Gene Delivery 435, 436, 491
Pharmacology/Toxicology 365 Non-Viral Vectorology 479
RNAi and shRNA 386, 625 Oligonucleotide Based Therapies 440
Oncolytic Virus 68
Transgene Engineering Physcial Delviery 491
AAV Vectors 456 Regulatory Issues 465, 600
Gene Expression 456 Respiratory/Lung Disorder 22
Hemophilia 456 RNA Viral Vectors 68
Tissue Engineering 491
Transposon
Chimeric Antigen Receptors 705 Vector Characterization/Biology
Non-Viral Gene Delivery 628, 705 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,
Tumor Microenvironment
541, 546, 547, 548, 607, 695, 727
Adenovirus Vectors 14, 17, 655
Animal Models 2
Immunotherapy 662
Cancer - Gene Therapy 409, 548
Tumor-Specific Promoter Cancer - Targeted Therapy 655
Cancer - Gene Therapy 677 Cell Reprogramming 344
Cancer - Targeted Therapy 677 Clinical Gene Therapy 547
Hematologic Diseases 677 Crispr/Cas9 293
DNA Viral Vectors 14, 17, 107, 108, 254, 727
Tunneling Nanotubes Epigenetic Regulation 8
Cell Delivery 336 Evolution and Development 389
Cell Therapy - Hematopoietic 336 Exosomes 594
Genetic Diseases 336 Foamy Virus 1
Gene Expression 10, 11, 91, 289, 474, 546
U1 Adaptors, iRGD, KRAS, MYC, Pancreatic Cancer Genetic Diseases 257
Cancer - Targeted Therapy 262 Hematologic Diseases 230, 473
Oligonucleotide Based Therapies 262 Hematopoietic Stem Cells 1
RNA Delivery 262 Hsv Vectors 107
Neurological Disorders 259 Humoral Immune Response 82
Neuromuscular Disorders 259 Insertional Mutagenesis 2
Oligonucleotide Based Therapies 259 Lentivirus Vectors 2, 230, 457, 709
Metabolic Diseases 218, 355
Ultrasound
Animal Models 492
Hemophilia 492
Non-Viral Gene Delivery 344, 355
Urea Cycle Disorder Ophthalmic Diseases 594, 607
AAV Vectors 45 Precision Medicine 7
Cell Therapy - ES Cells and iPS Cells 347 QC/QA Assays 34, 156, 473
Gene Correction/Modification/Targeting 347 Regulation 546
Genetic Diseases 45 Respiratory/Lung Disorders 181
Metabolic Diseases 347 RNA Viral Vectors 1, 409
Neurological Disorders 45 SERCaMP, ATV6, ER Stress 218
Single-Molecule, Real-Time Sequencing 7
Vaccines Targeted Gene Expression 108, 181, 298, 307, 478, 695
AAV Vectors 196, 722 Thermostability 34
Adenovirus Vectors 22, 36, 642 Vector Immunology/Host Responses 82
Cancer - Gene Therapy 643 Vector Production 9, 12, 156, 302, 304, 457, 709
Cancer - T-Cell Therapy 68, 191, 596 Vector Transduction 594
Cell Delivery 596 Viral Evolution 541
DNA Vaccines 436 Vector Immunology/Host Responses
DNA Viral Vectors 712 AAV Vectors 77, 78, 79, 80, 81, 82, 90, 185, 310, 378, 424,
Immunotherapy 191, 196, 395, 642, 643 467, 595, 696, 697, 698, 700, 701, 702
Infectious Diseases 22, 435, 440, 479 Adenovirus Vectors 517
Musculo-Skeletal Disorders 722 Animal Models 63, 415, 437, 698
Nanobiotechnology 600 Antibody Response 77

KEYWORD INDEX
Anti-Transgene 694 Lentivirus Vectors 286, 288, 457, 458, 464, 703, 704, 707,
Cancer - Gene Therapy 403 708, 709, 711
Cancer - Targeted Therapy 208, 415, 517 Orbital Shaken Bioreactors 463
CD8+ T Cells, Ovalbumin 79 Oversized Vector 92
Cell Therapy - Lung 438 QC/QA Assays 156, 290
Cell Therapy - Neuronal 208 Regulatory Issues 464, 465
Clinical Gene Therapy 185, 697 RNA Viral Vectors 6, 288, 703
Exosomes 595 Vaccines 465, 712
Gene Correction/Modification/Targeting 764 Vector Characterization/Biology 9, 12, 156, 302, 304, 457,
Gene Expression 310, 700, 701 709
Hemophilia 78 Vector Purification 294
Hsv Vectors 518 XLRP 101
Humoral Immune Response 82
Immunotherapy 51, 63, 81, 395, 518 Vector Purification
Infectious Diseases 79, 437 AAV Vectors 294
Lentivirus Vectors 51, 764 DNA Viral Vectors 294
Long Term Expression 424 Vector Production 294
Vector Transduction
Exosomes 594
Ophthalmic Diseases 80, 694
Pre-Existing AAV Antibodies 698
QC/QA Assays 467 Viral Evolution
Reovirus 415 AAV Vectors 541
Retrovirus Vectors 403 Vector Characterization/Biology 541
Tissue Engineering 702 Viral Transduction Enhancers
Tlr9 Signaling 696 Hematopoietic Stem Cells 536
Vaccines 395 Lentivirus Vectors 536
Vector Characterization/Biology 82 Nanobiotechnology 536
Vector Production Wet Age-Related Macular Degeneration

AAV Vectors 9, 12, 87, 92, 94, 95, 97, 100, 101, 290, 292, AAV Vectors 248
294, 302, 304, 308, 309, 460, 463, 471, 539, 549, 591, Clinical Gene Therapy 248
672, 684, 710 Ophthalmic Diseases 248
Adenovirus Vectors 706, 713
Cancer - Gene Therapy 672, 703 WT1
Capsid Modification 95 Cancer - T-Cell Therapy 193
Cell Processing 707 Chimeric Antigen Receptors 193
Cell Reprogramming 704 Immunotherapy 193
XIST-Mediated Chromosome Silencing
Chromatography 100
Continuous Flow Ultracentrifugation 97
Crude Lysate 290
Cystic Fibrosis 711 XLRP
Database Development 591 AAV Vectors 101
Design Of Experiment 708 Genetic Diseases 101
DNA Viral Vectors 87, 97, 294, 549, 712 Vector Production 101
Gen Expression 704
Gene Correction/Modification/Targeting 286 Zinc-Finger Nucleases
Gene Expression 292, 471 AAV Vectors 133, 485
Gene Therapy 94 Adenovirus Vectors 484
Genetic Diseases 101 Animal Models 38
Gmp Manufacturing 460, 710 Cancer - Cell Therapy 641
Good Manufacturing Procedures 100, 308, 309, 460, 463, Cancer - Gene Therapy 752
591, 706, 710, 713 Cancer - T-Cell Therapy 752
Hematopoietic Stem Cells 458 Cell Therapy - Hematopoietic 734
Hemophilia 92, 684 Gene Correction/Modification/Targeting 37, 38, 118, 127,
Hsv Vectors 94 130, 133, 315, 485, 563, 566, 641, 765
Hybrid Vector 672 Genetic Modification 734
Infectious Diseases 539 Hematologic Diseases 118
KEYWORD INDEX
Hematopoietic Stem Cells 37, 734
Mucopolysaccharidosis 485
Multiple Myeloma 752
XIST-Mediated Chromosome Silencing 315
β-Thalassemias, Hemoglobinopathies


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KEYWORD INDEX

Molecular Therapy Volume 24, Supplement 1, May 2016 S321

S322 Molecular Therapy Volume 24, Supplement 1, May 2016

Molecular Therapy Volume 24, Supplement 1, May 2016 S323

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S326 Molecular Therapy Volume 24, Supplement 1, May 2016

Molecular Therapy Volume 24, Supplement 1, May 2016 S327

S328 Molecular Therapy Volume 24, Supplement 1, May 2016

Gene Expression Gene Therapy

S334 Molecular Therapy Volume 24, Supplement 1, May 2016

Molecular Therapy Volume 24, Supplement 1, May 2016 S335

Molecular Therapy Volume 24, Supplement 1, May 2016 S337

S338 Molecular Therapy Volume 24, Supplement 1, May 2016

Luekocyte Adhesion Deficiency Metabolic Disease

Lung Cancer Metabolic Diseases

Molecular Therapy Volume 24, Supplement 1, May 2016 S339

mRNA Mutated Neoantigens

Neurological Disorders Neuromuscular Disorders

S342 Molecular Therapy Volume 24, Supplement 1, May 2016

Oncolytic Virus Optogenetics

Ophthalmic Diseases Oversized Vector

Molecular Therapy Volume 24, Supplement 1, May 2016 S343

S344 Molecular Therapy Volume 24, Supplement 1, May 2016

Regulatory T Cell RNA Delivery

Molecular Therapy Volume 24, Supplement 1, May 2016 S345

S346 Molecular Therapy Volume 24, Supplement 1, May 2016

Targeted Gene Expression T-Cells

S348 Molecular Therapy Volume 24, Supplement 1, May 2016

Vector Production Wet Age-Related Macular Degeneration

S350 Molecular Therapy Volume 24, Supplement 1, May 2016

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