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FRONTIERS IN IMMUNOLOGY, ISSN 1664-3224, 03/2014, Volume 5
Journal Article
Frontiers in Immunology, ISSN 1664-3224, 2014, Volume 5, pp. 82 - 82
Alipogene tiparvovec (Glybera(®)) is an adeno-associated virus serotype 1 (AAV1)-based gene therapy that has been developed for the treatment of patients with... 
Alipogene tiparvovec | Clinical safety | Immune responses | Gene therapy | Adeno-associated viral vectors | Clinical efficacy | alipogene tiparvovec | adeno associated viral vectors | clinical efficacy | Gene Therapy | clinical safety
Journal Article
Molecular Therapy, ISSN 1525-0016, 09/2013, Volume 21, Issue 9, pp. 1727 - 1737
Journal Article
Journal Article
Frontiers in Immunology, 02/2014, Volume 5
Transitioning to human trials from preclinical models resulted in the emergence of inhibitory AAV vector immune responses which has become a hurdle for... 
preclinical models | aav | adeno-associated virus | Immune responses | inhibitory AAV
Journal Article
Human Gene Therapy, ISSN 1043-0342, 11/2017, Volume 28, Issue 11, pp. 161 - 1074
Journal Article
Molecular Therapy - Methods & Clinical Development, ISSN 2329-0501, 2016, Volume 3, Issue C, pp. 16063 - 16063
Hemophilia A and B are coagulation disorders resulting from the loss of functional coagulation factor VIII (FVIII) or factor IX proteins, respectively. Gene... 
MEDICINE, RESEARCH & EXPERIMENTAL | UNFOLDED PROTEIN RESPONSE | B-DOMAIN | COAGULATION-FACTOR-VIII | HEMOPHILIA-A | FACTOR-IX | ENDOTHELIAL-CELLS | GENE-THERAPY | ENDOPLASMIC-RETICULUM STRESS | IMMUNE TOLERANCE | VON-WILLEBRAND-FACTOR
Journal Article
Journal Article
Proceedings of the National Academy of Sciences of the United States, ISSN 0027-8424, 03/2011, Volume 108, Issue 9, p. 3743
Because our in silico analysis with a human transcription factor database demonstrated the presence of several binding sites for NF-[kappa]B, a central... 
Transcription factors | Immune response | Genetic aspects | Dependoviruses | Research | Gene expression | Health aspects | Proteins | Signal transduction | Molecules | Cytokines | Viruses | Gene therapy | Binding sites
Journal Article
Gene Therapy, ISSN 0969-7128, 09/2007, Volume 14, Issue 17, pp. 1249 - 1260
Using murine models, we have previously demonstrated that recombinant adeno-associated virus (rAAV)-mediated microdystrophin gene transfer is a promising... 
MEDICINE, RESEARCH & EXPERIMENTAL | LONG-TERM CORRECTION | gene transfer | BIOCHEMISTRY & MOLECULAR BIOLOGY | TRANSDUCTION | T-CELL | MDX MICE | immune response | BIOTECHNOLOGY & APPLIED MICROBIOLOGY | AAV | Duchenne muscular dystrophy | HEMOPHILIA-B | FACTOR-IX | IN-VIVO | GENETICS & HEREDITY | ADENOASSOCIATED VIRUS VECTORS | DUCHENNE MUSCULAR-DYSTROPHY | skeletal muscle | dog | DIRECTED GENE-THERAPY | Calmodulin - genetics | Dependovirus - genetics | Species Specificity | Parvoviridae Infections - immunology | Molecular Sequence Data | RNA, Messenger - analysis | Genetic Vectors - adverse effects | Muscle Fibers, Skeletal - virology | Muscle, Skeletal - immunology | Base Sequence | Muscular Dystrophy, Duchenne - immunology | Models, Animal | Muscular Dystrophy, Animal - therapy | Muscular Dystrophy, Duchenne - therapy | Transgenes | Dystrophin - metabolism | Transduction, Genetic - methods | Immunosuppressive Agents - administration & dosage | T-Lymphocytes, Cytotoxic - immunology | Muscular Dystrophy, Animal - immunology | Lymphocyte Activation | Mice, Inbred C57BL | Injections, Intramuscular | Muscle Fibers, Skeletal - immunology | Reverse Transcriptase Polymerase Chain Reaction | Genetic Vectors - genetics | Animals | Genetic Engineering | Dystrophin - genetics | Interferon-gamma - immunology | Cyclosporine - administration & dosage | Dogs | Mice | beta-Galactosidase - genetics | Genetic Therapy - adverse effects | Genetic Therapy - methods | Immune response | Genetic transformation | Physiological aspects | Muscles | Genetic aspects | Dependoviruses | Research | Health aspects | Signal transduction | Gene therapy | Adenoviruses | Muscular dystrophy | Immune system | Index Medicus
Journal Article
Cellular Immunology, ISSN 0008-8749, 08/2019, Volume 342, pp. 103722 - 103722
Although gene transfer using adeno-associated virus (AAV) vectors has made tremendous progress in recent years, challenges remain due to vector-specific... 
Adeno-associated virus vectors | T and B cells | Immune responses | NEUTRALIZING ANTIBODIES | IMMUNOLOGY | VIRAL VECTORS | CD8+T CELLS | CELL BIOLOGY | T-CELL RESPONSES | HUMAN HEPATOCYTES | IMMUNE-RESPONSES | FACTOR-IX | ADENOASSOCIATED VIRUS VECTORS | HUMORAL IMMUNITY | GENE-TRANSFER | Index Medicus
Journal Article
Blood, ISSN 0006-4971, 03/2013, Volume 121, Issue 12, pp. 2224 - 2233
Journal Article