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1. Full Text Unraveling the Complex Story of Immune Responses to AAV Vectors Trial After Trial
by Vandamme, Céline and Adjali, Oumeya and Mingozzi, Federico
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
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2. Full Text Safety and Long-Term Efficacy of AAV4 Gene Therapy in Patients with RPE65 Leber Congenital Amaurosis
by Le Meur, Guylène and Lebranchu, Pierre and Billaud, Fanny and Adjali, Oumeya and Schmitt, Sébastien and Bézieau, Stéphane and Péréon, Yann and Valabregue, Romain and Ivan, Catherine and Darmon, Christophe and Moullier, Philippe and Rolling, Fabienne and Weber, Michel
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
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3. Full Text Long-term microdystrophin gene therapy is effective in a canine model of Duchenne muscular dystrophy
by Le Guiner, Caroline and Servais, Laurent and Montus, Marie and Larcher, Thibaut and Fraysse, Bodvaël and Moullec, Sophie and Allais, Marine and François, Virginie and Dutilleul, Maeva and Malerba, Alberto and Koo, Taeyoung and Thibaut, Jean-Laurent and Matot, Béatrice and Devaux, Marie and Le Duff, Johanne and Deschamps, Jack-Yves and Barthelemy, Inès and Blot, Stéphane and Testault, Isabelle and Wahbi, Karim and Ederhy, Stéphane and Martin, Samia and Veron, Philippe and Georger, Christophe and Athanasopoulos, Takis and Masurier, Carole and Mingozzi, Federico and Carlier, Pierre and Gjata, Bernard and Hogrel, Jean-Yves and Adjali, Oumeya and Mavilio, Fulvio and Voit, Thomas and Moullier, Philippe and Dickson, George
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
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4. Full Text Five Years of Successful Inducible Transgene Expression Following Locoregional Adeno-Associated Virus Delivery in Nonhuman Primates with No Detectable Immunity
by Guilbaud, Mickaël and Devaux, Marie and Couzinié, Celia and Le Duff, Johanne and Toromanoff, Alice and Vandamme, Céline and Jaulin, Nicolas and Gernoux, Gwladys and Larcher, Thibaut and Moullier, Philippe and Le Guiner, Caroline and Adjali, Oumeya
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
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5. Full Text Forelimb Treatment in a Large Cohort of Dystrophic Dogs Supports Delivery of a Recombinant AAV for Exon Skipping in Duchenne Patients
by Le Guiner, Caroline and Montus, Marie and Servais, Laurent and Cherel, Yan and Francois, Virginie and Thibaud, Jean-Laurent and Wary, Claire and Matot, Béatrice and Larcher, Thibaut and Guigand, Lydie and Dutilleul, Maeva and Domenger, Claire and Allais, Marine and Beuvin, Maud and Moraux, Amélie and Le Duff, Johanne and Devaux, Marie and Jaulin, Nicolas and Guilbaud, Mickaël and Latournerie, Virginie and Veron, Philippe and Boutin, Sylvie and Leborgne, Christian and Desgue, Diana and Deschamps, Jack-Yves and Moullec, Sophie and Fromes, Yves and Vulin, Adeline and Smith, Richard H and Laroudie, Nicolas and Barnay-Toutain, Frédéric and Rivière, Christel and Bucher, Stéphanie and Le, Thanh-Hoa and Delaunay, Nicolas and Gasmi, Mehdi and Kotin, Robert M and Bonne, Gisèle and Adjali, Oumeya and Masurier, Carole and Hogrel, Jean-Yves and Carlier, Pierre and Moullier, Philippe and Voit, Thomas
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
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6. Full Text Intrathymic adeno-associated virus gene transfer rapidly restores thymic function and long-term persistence of gene-corrected T cells
by Pouzolles, Marie and Machado, Alice and Guilbaud, Mickaël and Irla, Magali and Gailhac, Sarah and Barennes, Pierre and Cesana, Daniela and Calabria, Andrea and Benedicenti, Fabrizio and Sergé, Arnauld and Raman, Indu and Li, Quan-Zhen and Montini, Eugenio and Klatzmann, David and Adjali, Oumeya and Taylor, Naomi and Zimmermann, Valérie S
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
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7. Full Text Stability of the adeno-associated virus 8 reference standard material
by Penaud-Budloo, Magalie and Broucque, Frédéric and Harrouet, Katell and Bouzelha, Mohammed and Saleun, Sylvie and Douthe, Sandy and D’Costa, Susan and Ogram, Sushma and Adjali, Oumeya and Blouin, Véronique and Lock, Martin and Snyder, Richard O and Ayuso, Eduard
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
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8. Full Text Liver-Specific Transcriptional Modules Identified by Genome-Wide In Silico Analysis Enable Efficient Gene Therapy in Mice and Non-Human Primates
by Chuah, Marinee K and Petrus, Inge and De Bleser, Pieter and Le Guiner, Caroline and Gernoux, Gwladys and Adjali, Oumeya and Nair, Nisha and Willems, Jessica and Evens, Hanneke and Rincon, Melvin Y and Matrai, Janka and Di Matteo, Mario and Samara-Kuko, Ermira and Yan, Bing and Acosta-Sanchez, Abel and Meliani, Amine and Cherel, Ghislaine and Blouin, Véronique and Christophe, Olivier and Moullier, Philippe and Mingozzi, Federico and VandenDriessche, Thierry
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
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