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1. Full Text CD8+ T Cell Recognition of Epitopes Within the Capsid of Adeno-associated Virus 8–based Gene Transfer Vectors Depends on Vectors’ Genome
by Wu, Te-Lang and Li, Hua and Faust, Susan M and Chi, Emily and Zhou, Shangzhen and Wright, Fraser and High, Katherine A and Ertl, Hildegund CJ
Molecular Therapy, ISSN 1525-0016, 01/2014, Volume 22, Issue 1, pp. 42 - 51
Self-complementary adeno-associated viral (AAV) vectors expressing human factor IX (hF.IX) have achieved transient or sustained correction of hemophilia B in... 
EFFICIENT TRANSDUCTION | MEDICINE, RESEARCH & EXPERIMENTAL | AAV VECTORS | EFFICACY | SAFETY | HUMAN FIX | SEROTYPE | BIOTECHNOLOGY & APPLIED MICROBIOLOGY | IMMUNE-RESPONSES | FACTOR-IX | GENETICS & HEREDITY | MICE | LIVER TRANSDUCTION | Dependovirus - genetics | Transduction, Genetic | Genetic Vectors - immunology | Humans | Dependovirus - immunology | Male | Epitopes, T-Lymphocyte | Genetic Vectors - standards | Genetic Vectors - genetics | Capsid - immunology | Lymphocyte Activation - immunology | Animals | Capsid Proteins - immunology | T-Cell Antigen Receptor Specificity | Immunologic Memory | Mice | CD8-Positive T-Lymphocytes - immunology | Genome | Quality Control | Original
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2. Full Text CRISPR/Cas9‐mediated genome engineering: An adeno‐associated viral (AAV) vector toolbox
by Senís, Elena and Fatouros, Chronis and Große, Stefanie and Wiedtke, Ellen and Niopek, Dominik and Mueller, Ann‐Kristin and Börner, Kathleen and Grimm, Dirk
Biotechnology Journal, ISSN 1860-6768, 11/2014, Volume 9, Issue 11, pp. 1402 - 1412
Its remarkable ease and efficiency make the CRISPR (clustered regularly interspaced short palindromic repeats) DNA editing machinery highly attractive as a new... 
CRISPR | Viral vectors | Genome engineering | AAV | Gene editing | CELLS | IMMUNITY | BIOCHEMICAL RESEARCH METHODS | CAS SYSTEMS | GUIDE | ENDONUCLEASE | BIOTECHNOLOGY & APPLIED MICROBIOLOGY | LIVER | GENE-THERAPY | MUTATIONS | VIRUS VECTORS | Genetic Engineering - methods | Dependovirus - genetics | Animals | Base Sequence | Humans | HEK293 Cells | Molecular Sequence Data | Mice | MicroRNAs - genetics | Binding Sites | CRISPR-Cas Systems - genetics | Genetic Vectors - genetics | Genetic vectors | RNA | Genomics | Genetically modified organisms | Genetic engineering | Cross infection | Nosocomial infections | T cells | Health aspects
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3. Full Text Immune responses to gene therapy vectors: influence on vector function and effector mechanisms
by Bessis, N and Bessis, N and GarciaCozar, F.J and Boissier, M.-C and Boissier, M.-C
Gene Therapy, ISSN 0969-7128, 10/2004, Volume 11, Issue S1, pp. S10 - S17
Circumventing the immune response to the vector is a major challenge with all vector types. Viral vectors are the most likely to induce an immune response,... 
Innate immunity | Specific immunity | AAV | MEDICINE, RESEARCH & EXPERIMENTAL | HUMAN DENDRITIC CELLS | RECOMBINANT ADENOASSOCIATED VIRUS | BIOCHEMISTRY & MOLECULAR BIOLOGY | CD40 LIGAND | ADENOVIRAL VECTORS | CYTOTOXIC T-LYMPHOCYTES | innate immunity | BIOTECHNOLOGY & APPLIED MICROBIOLOGY | IN-VIVO | PLASMID DNA | TRANSIENT IMMUNOSUPPRESSION | FACTOR-IX | GENETICS & HEREDITY | TRANSGENE PRODUCT | specific immunity | Immune System - physiology | Genetic Vectors - administration & dosage | Genetic Vectors - immunology | Humans | DNA, Bacterial - immunology | Immunosuppression | Animals | Leukocytes, Mononuclear - immunology | Adenoviridae - genetics | Antibodies, Bacterial - immunology | Adenoviridae - immunology | Transduction, Genetic - methods | Cytokines - immunology | Genetic Therapy - adverse effects | Genetic Therapy - methods | Physiological aspects | Immune response | Genetic vectors | Gene therapy | Health aspects
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4. Full Text Corticospinal tract transduction: a comparison of seven adeno-associated viral vector serotypes and non-integrating lentiviral vector
by Hutson, T.H and Verhaagen, J and Yanez-Munoz, R.J and Moon, L.D
Gene Therapy, ISSN 0969-7128, 2012, Volume 19, Issue 1, pp. 49 - 60
The corticospinal tract (CST) is extensively used as a model system for assessing potential therapies to enhance neuronal regeneration and functional recovery... 
spinal cord injury | AAV | corticospinal tract | transduction | integration-deficient lentiviral vector | MOUSE-BRAIN | MEDICINE, RESEARCH & EXPERIMENTAL | PYRAMIDAL TRACT | NEUROTROPHIC FACTOR | AXON-REGENERATION | BIOCHEMISTRY & MOLECULAR BIOLOGY | EFFICIENT GENE DELIVERY | CORTICAL-NEURONS | BIOTECHNOLOGY & APPLIED MICROBIOLOGY | GENETICS & HEREDITY | SPINAL-CORD-INJURY | CENTRAL-NERVOUS-SYSTEM | VESICULAR STOMATITIS-VIRUS | PARKINSONS-DISEASE | Dependovirus - genetics | Genetic Therapy | Microglia - metabolism | Cell Count | Humans | Neurons - cytology | Lentivirus - metabolism | Transfection - methods | HEK293 Cells | Pyramidal Tracts - metabolism | Lentivirus - genetics | Neurons - metabolism | Serotyping | Astrocytes - cytology | Microglia - cytology | Gene Transfer Techniques | Green Fluorescent Proteins - metabolism | Dependovirus - metabolism | Genetic Vectors - metabolism | Rats | Dependovirus - classification | Virus Integration | Rats, Sprague-Dawley | Genetic Vectors - genetics | Animals | Pyramidal Tracts - cytology | Astrocytes - metabolism | Care and treatment | Neurons | Physiological aspects | Spinal cord injuries | Research | Gene therapy | Health aspects | Risk factors | Comparative studies | Transducers | Models | Vector space | Tropism | Regeneration | Central nervous system | Green fluorescent protein | Oligodendrocytes | Recovery of function | Serotypes | Pyramidal tracts | Spinal cord injury | Expression vectors
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5. Full Text Viral vector-mediated gene therapies
by Hollinger, Katrin and Chamberlain, Jeffrey S
Current Opinion in Neurology, ISSN 1350-7540, 10/2015, Volume 28, Issue 5, pp. 522 - 527
Purpose of review Gene therapy as a treatment for neuromuscular disease has significantly advanced over the past decade. In the present review, the progress of... 
Cas9/CRISPR | Adeno-associated viruses | Dystrophin | Duchenne muscular dystrophy | CELLS | MUSCULAR-DYSTROPHY | dystrophin | MODEL | NEUROSCIENCES | CLINICAL NEUROLOGY | RESPONSES | adeno-associated viruses | VASCULAR DELIVERY | AAV | MUSCLE FUNCTION | MICE | EXPRESSION | Dependovirus - genetics | Genetic Vectors - therapeutic use | Muscular Dystrophy, Duchenne - therapy | Humans | Genetic Therapy - methods | CRISPR | Cas9
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6. Full Text Pre-existing antibodies to candidate gene therapy vectors (adeno-associated vector serotypes) in domestic cats
by Li, Pengfei and Boenzli, Eva and Hofmann-Lehmann, Regina and Katrin Helfer-Hungerbuehler, A
PLoS ONE, ISSN 1932-6203, 03/2019, Volume 14, Issue 3, p. e0212811
Adeno-associated virus (AAV) vectors represent promising candidates for gene therapy; however, pre-existing neutralizing antibodies (NAb) may reduce AAV vector... 
AAV VECTORS | NEUTRALIZING ANTIBODIES | SAFETY | IMMUNE-RESPONSES | MULTIDISCIPLINARY SCIENCES | FACTOR-IX | LIVER | HUMORAL IMMUNITY | ANIMAL-MODELS | TRANSDUCTION | PHASE-I | Cats | Usage | Genetic vectors | Analysis | Outcome and process assessment (Health Care) | Genetic research | Genetic aspects | Dependoviruses | Gene therapy | Health aspects | Viral antibodies | Genes | Antibodies | Biopharmaceutics | Animal models | Immunoglobulins | Congenital diseases | Disease | Laboratories | Galactosidase | Clinical trials | Viruses | Monkeys & apes | Vectors (Biology) | Studies | Serotypes | In vivo methods and tests | Expression vectors
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7. Full Text Vector design influences hepatic genotoxicity after adeno-associated virus gene therapy
by Chandler, Randy J and La Fave, Matthew C and Varshney, Gaurav K and Trivedi, Niraj S and Carrillo-Carrasco, Nuria and Senac, Julien S and Wu, Weiwei and Hoffmann, Victoria and Elkahloun, Abdel G and Burgess, Shawn M and Venditti, Charles P
Journal of Clinical Investigation, ISSN 0021-9738, 02/2015, Volume 125, Issue 2, pp. 870 - 880
The use of adeno-associated virus (AAV) as a gene therapy vector has been approved recently for clinical use and has demonstrated efficacy in a growing number... 
MEDICINE, RESEARCH & EXPERIMENTAL | AAV VECTORS | MURINE MODEL | HEPATOCELLULAR-CARCINOMA | INTEGRATION SITES | MOUSE MODEL | LIVER | LARGE-SCALE | INSERTIONAL MUTAGENESIS | METHYLMALONIC ACIDEMIA | LEBERS CONGENITAL AMAUROSIS | Transduction, Genetic | Liver Neoplasms - genetics | Pregnancy Proteins - genetics | Liver Neoplasms - therapy | Pregnancy Proteins - metabolism | Dependovirus | Animals | Mice, Mutant Strains | Carcinoma, Hepatocellular - genetics | Liver Neoplasms - metabolism | Carcinoma, Hepatocellular - therapy | Mice | Genetic Vectors | Carcinoma, Hepatocellular - metabolism | Genetic Therapy - methods | Usage | Liver diseases | Genetic vectors | Patient outcomes | Dependoviruses | Gene therapy | Properties | Risk factors | Studies | Rodents | Liver | MicroRNAs | Software | Genomes | Gene expression | Tumors
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8. Full Text Viral vectors: from virology to transgene expression
by Bouard, D and Alazard-Dany, N and Cosset, F-L
British Journal of Pharmacology, ISSN 0007-1188, 05/2009, Volume 157, Issue 2, pp. 153 - 165
In the late 1970s, it was predicted that gene therapy would be applied to humans within a decade. However, despite some success, gene therapy has still not... 
vectors production | AAV | cell targeting | gene therapy | HSV | pseudotyping | lentivectors | transcriptional targeting | viral vectors | Lentivectors | Ad | Vectors production | Transcriptional targeting | Viral vectors | Gene therapy | Pseudotyping | Cell targeting | EFFICIENT GENE-TRANSFER | SEVERE COMBINED IMMUNODEFICIENCY | RECOMBINANT ADENOASSOCIATED VIRUS | ENGINEERED LENTIVIRAL VECTORS | THYMIDINE KINASE GENE | DEPENDENT ADENOVIRAL VECTORS | HSV-1 AMPLICON VECTORS | MURINE LEUKEMIA-VIRUS | TARGETED RETROVIRAL VECTORS | PHARMACOLOGY & PHARMACY | TRANSDUCE AIRWAY EPITHELIA | Genetic Therapy | Animals | Viruses - genetics | Genetic Vectors | Transgenes | Humans | Reviews | Themed Section
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9. Full Text Viral vector-based improvement of optic nerve regeneration: Characterization of individual axons' growth patterns and synaptogenesis in a visual target
by Yungher, B.J and Luo, X and Salgueiro, Y and Blackmore, M.G and Park, K.K
Gene Therapy, ISSN 0969-7128, 10/2015, Volume 22, Issue 10, pp. 811 - 821
Lack of axon growth ability in the central nervous system poses a major barrier to achieving functional connectivity after injury. Thus, a non-transgenic... 
RNA INTERFERENCE | MEDICINE, RESEARCH & EXPERIMENTAL | GANGLION-CELL AXONS | NEUROTROPHIC FACTOR | BIOCHEMISTRY & MOLECULAR BIOLOGY | AAV-MEDIATED EXPRESSION | SUPERIOR COLLICULUS | BIOTECHNOLOGY & APPLIED MICROBIOLOGY | IN-VIVO | GENETICS & HEREDITY | CORTICOSPINAL TRACT | SPINAL-CORD-INJURY | ADULT HAMSTERS | GENE-THERAPY | Nerve Regeneration - genetics | Neurogenesis - genetics | Optic Nerve Injuries - genetics | PTEN Phosphohydrolase - genetics | RNA, Small Interfering - genetics | Optic Nerve - physiology | Animals | Mice | Dependovirus | Genetic Vectors | RNA, Small Interfering - administration & dosage | Optic Nerve Injuries - therapy | Regeneration | Growth | Physiological aspects | Nervous system | Genetic aspects | Research | Ciliary neurotrophic factor | Cellular biology | Injuries | PTEN | Axon injury | Axon guidance | Axon regeneration | Retinal ganglion cell
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10. Full Text Viral Vectors for Gene Therapy: Translational and Clinical Outlook
by Kotterman, Melissa A and Chalberg, Thomas W and Schaffer, David V
Annual Review of Biomedical Engineering, ISSN 1523-9829, 12/2015, Volume 17, Issue 1, pp. 63 - 89
In a range of human trials, viral vectors have emerged as safe and effective delivery vehicles for clinical gene therapy, particularly for monogenic recessive... 
genotoxicity | retrovirus | adeno-associated virus | vector engineering | adenovirus | lentivirus | Retrovirus | Adeno-Associated virus | Vector engineering | Adenovirus | Lentivirus | Genotoxicity | INTEGRATION SITE SELECTION | TARGETED ONCOLYTIC POXVIRUS | ENGINEERING, BIOMEDICAL | DIRECTED EVOLUTION | RETROVIRAL VECTORS | CALCIUM UP-REGULATION | MOUSE MODEL | ADENOASSOCIATED VIRUS AAV | PHASE-I | CYSTIC-FIBROSIS GENE | PARKINSONS-DISEASE | Dependovirus - genetics | Immunologic Deficiency Syndromes - therapy | Genetic Engineering | Retroviridae - genetics | Humans | Translational Medical Research | Lentivirus - genetics | Clinical Trials as Topic | Genetic Vectors | Genetic Therapy - methods | Genetic vectors | Genetic aspects | Host-virus relationships | Gene therapy | Observations | Genetic translation | Methods
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