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Human gene therapy, ISSN 1043-0342, 11/2017, Volume 28, Issue 11, pp. 161 - 1074
Review Articles | gene therapy | AAV vectors | T cells | antibody responses | immune responses | Genetics & Heredity | Life Sciences & Biomedicine | Biotechnology & Applied Microbiology | Medicine, Research & Experimental | Science & Technology | Research & Experimental Medicine | Capsid Proteins - therapeutic use | Dependovirus - genetics | Humans | Dependovirus - immunology | Genetic Vectors - adverse effects | Clinical Trials as Topic | Genetic Therapy - trends | Genetic Vectors - therapeutic use | Capsid Proteins - adverse effects | Immunity, Cellular - immunology | Capsid Proteins - immunology | CD8-Positive T-Lymphocytes - immunology | Genetic Therapy - adverse effects | Medical research | Antigen presentation | Antigens | Immunological memory | Memory cells | Immune response | Gene transfer | Toxicity | CD8 antigen | Liver | Clinical trials | Antibodies | Viruses | Lymphocytes T | Gene expression | Tissues | Human tissues | Immunogenicity | Lymphocytes B | Lymphocytes | Index Medicus | Life Sciences | Human health and pathology | Review
Journal Article
Molecular therapy, ISSN 1525-0016, 01/2018, Volume 26, Issue 1, pp. 256 - 268
clinical trial | AAV vector | gene therapy | inherited retinal dystrophies | RPE65 | Immunoglobulins | Visual field | Cortex | Visual pathways | Retina | Genomes | Inflammation | Patients | Vectors (Biology) | Visual perception | Acuity | Studies | Immunology | Lighting | Blindness | Questionnaires | Nystagmus | Functional magnetic resonance imaging | Retinal degeneration | Serotypes | Photoreceptors | Mutation | Gene therapy | Index Medicus | Life Sciences | Human health and pathology | Original
Journal Article
Nature communications, ISSN 2041-1723, 07/2017, Volume 8, Issue 1, pp. 16105 - 16105
Science & Technology - Other Topics | Multidisciplinary Sciences | Science & Technology | Gene Transfer Techniques | Genetic Therapy | Muscular Dystrophy, Animal - genetics | Muscular Dystrophy, Animal - physiopathology | Male | Muscle, Skeletal - metabolism | Dependovirus | Administration, Intravenous | Animals | Dystrophin - genetics | Muscular Dystrophy, Duchenne - physiopathology | Muscle, Skeletal - physiopathology | Dogs | Muscular Dystrophy, Animal - metabolism | Muscular Dystrophy, Animal - therapy | Muscular Dystrophy, Duchenne - metabolism | Muscular Dystrophy, Duchenne - genetics | Genetic Vectors | Transgenes | Disease Models, Animal | Medical research | Intravenous administration | Toxicity | Clinical trials | Muscles | Viruses | Muscular dystrophy | Skeletal muscle | Immunosuppression | Duchenne's muscular dystrophy | Dystrophy | Mutation | Gene therapy | Dystrophin | Expression vectors | Index Medicus | Life Sciences | Molecular biology | Biochemistry, Molecular Biology
Journal Article
Human gene therapy, ISSN 1043-0342, 07/2019, Volume 30, Issue 7, pp. 82 - 813
Research Articles | locoregional delivery | nonhuman primate | gene transfer | AAV | long-term follow-up | immunogenicity | Genetics & Heredity | Life Sciences & Biomedicine | Biotechnology & Applied Microbiology | Medicine, Research & Experimental | Science & Technology | Research & Experimental Medicine | Dependovirus - genetics | Gene Transfer Techniques | Genome, Viral | Gene Expression | Cytokines - metabolism | Follow-Up Studies | Genetic Vectors - administration & dosage | Macaca fascicularis | Dependovirus - immunology | Genetic Vectors - adverse effects | Muscle, Skeletal - metabolism | Immunity | Genetic Vectors - genetics | Animals | Time Factors | Antibodies, Viral - immunology | Muscle, Skeletal - pathology | Transgenes | Animal models | Immune response | Intravenous administration | Gene transfer | Muscles | Viruses | Monkeys | Vectors | Gene expression | Skeletal muscle | Immunogenicity | Primates | Immune system | Index Medicus | s
Journal Article
Molecular therapy, ISSN 1525-0016, 11/2014, Volume 22, Issue 11, pp. 1923 - 1935
Genetics & Heredity | Life Sciences & Biomedicine | Biotechnology & Applied Microbiology | Medicine, Research & Experimental | Science & Technology | Research & Experimental Medicine | Dependovirus - genetics | Genetic Therapy | Genetic Vectors - administration & dosage | Exons | Humans | Dose-Response Relationship, Drug | Forelimb - physiopathology | Animals | Dystrophin - genetics | Muscular Dystrophy, Duchenne - physiopathology | RNA, Small Nuclear - genetics | Dogs | Infusions, Intravenous | Muscular Dystrophy, Duchenne - genetics | RNA, Small Nuclear - metabolism | Muscular Dystrophy, Duchenne - therapy | Cohort Studies | Disease Models, Animal | Index Medicus | Life Sciences | Original
Journal Article
Journal of allergy and clinical immunology, ISSN 0091-6749, 02/2020, Volume 145, Issue 2, pp. 679 - 697.e5
zeta-associated protein of 70kDa | T-cell reconstitution | thymus | intrathymic gene transfer | medulla formation | humoral immunity | Severe combined immunodeficiency | gene therapy | Allergy | Immunology | Life Sciences & Biomedicine | Science & Technology | Animal models | Transplants & implants | Copy number | Epithelial cells | Stem cell transplantation | Genomes | Lymphocytes T | ZAP-70 protein | Hematopoietic stem cells | Thymus | Proteins | T-cell receptor | Efficiency | Lymphocytes | Transgenes | Antigens | Immunoglobulins | Integration | Autoantibodies | Immunodeficiency | Medulla | Gene expression | Vectors (Biology) | Hemopoiesis | Thymopoiesis | Mutagenesis | Serotypes | Mutation | Gene therapy | Laboratory animals | Index Medicus | Abridged Index Medicus | Life Sciences
Journal Article
Gene therapy, ISSN 0969-7128, 05/2019, Volume 26, Issue 5, pp. 211 - 215
Biochemistry & Molecular Biology | Life Sciences & Biomedicine | Genetics & Heredity | Biotechnology & Applied Microbiology | Medicine, Research & Experimental | Science & Technology | Research & Experimental Medicine | Dependovirus - genetics | Genetic Vectors - chemistry | Capsid Proteins - metabolism | Humans | Genetic Vectors - standards | Cryopreservation - standards | Dependovirus - physiology | Reference Standards | Dependovirus - chemistry | Virus Replication | Capsid Proteins - chemistry | HEK293 Cells | Protein Stability | Practice Guidelines as Topic | Sodium lauryl sulfate | Working groups | Capsid protein | Laboratories | Gel electrophoresis | Clinical trials | Genomes | Drug screening | Storage conditions | American Type Culture Collection | Titration | Gene therapy | Enzyme-linked immunosorbent assay | Expression vectors | Index Medicus | Life Sciences
Journal Article
Molecular therapy, ISSN 1525-0016, 09/2014, Volume 22, Issue 9, pp. 1605 - 1613
Genetics & Heredity | Life Sciences & Biomedicine | Biotechnology & Applied Microbiology | Medicine, Research & Experimental | Science & Technology | Research & Experimental Medicine | Liver - virology | Dependovirus - genetics | Computational Biology - methods | Genetic Vectors - administration & dosage | Humans | Liver - metabolism | Factor IX - genetics | Transcription Factors - genetics | Macaca - genetics | Organ Specificity | Transcription Factors - metabolism | Macaca - virology | Animals | Regulatory Elements, Transcriptional | Base Sequence | Conserved Sequence | Factor IX - metabolism | Mice | Genome | Binding Sites | Index Medicus | Life Sciences | Original
Journal Article