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1. 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
The aim of this study was the evaluation of the safety and efficacy of unilateral subretinal injection of the adeno-associated vector (AAV) serotypes 2 and 4... 
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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2. 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
Duchenne muscular dystrophy (DMD) is an incurable X-linked muscle-wasting disease caused by mutations in the dystrophin gene. Gene therapy using highly... 
ADENOASSOCIATED VIRAL VECTORS | SKELETAL-MUSCLE | HEMOPHILIA-B | MULTIDISCIPLINARY SCIENCES | IMMUNE-SYSTEM DEVELOPMENT | IN-VIVO | 0.9-PERCENT SALINE | EXPRESSION | MDX MICE | VIRUS VECTOR | DELIVERY | 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 | Recombinant | Expression vectors | Index Medicus | Life Sciences | Molecular biology | Biochemistry, Molecular Biology
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3. 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
Duchenne muscular dystrophy (DMD) is a severe muscle-wasting disorder caused by mutations in the dystrophin gene, without curative treatment yet available. Our... 
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 | Original
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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
Anti-transgene immune responses elicited after intramuscular (i.m.) delivery of recombinant adeno-associated virus (rAAV) have been shown to hamper long-term... 
Research Articles | MEDICINE, RESEARCH & EXPERIMENTAL | AAV-FACTOR-IX | LONG-TERM CORRECTION | CANINE MODEL | gene transfer | nonhuman primate | MEDIATED GENE-TRANSFER | VIRAL VECTORS | immunogenicity | HEMOPHILIA-B DOGS | SKELETAL-MUSCLE | locoregional delivery | LIPOPROTEIN-LIPASE DEFICIENCY | T-CELL RESPONSES | FACTOR-IX EXPRESSION | BIOTECHNOLOGY & APPLIED MICROBIOLOGY | AAV | long-term follow-up | GENETICS & HEREDITY | Animal models | Immune response | Intravenous administration | Gene transfer | Muscles | Viruses | Monkeys | Vectors | Gene expression | Immunity | Skeletal muscle | Immune systems | Immunogenicity | Primates | Recombinant | s
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5. 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
Adeno-associated virus (AAV) vectors are extensively used for gene therapy clinical trials. Accurate and standardized titration methods are essential for... 
MEDICINE, RESEARCH & EXPERIMENTAL | VIRAL VECTOR | BIOTECHNOLOGY & APPLIED MICROBIOLOGY | BIOCHEMISTRY & MOLECULAR BIOLOGY | GENETICS & HEREDITY | Working groups | Capsid protein | Laboratories | Storage conditions | Clinical trials | American Type Culture Collection | Titration | Genomes | Drug screening | Gene therapy | Enzyme-linked immunosorbent assay | Expression vectors
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6. 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
The robustness and safety of liver-directed gene therapy can be substantially improved by enhancing expression of the therapeutic transgene in the liver. To... 
MEDICINE, RESEARCH & EXPERIMENTAL | SEQUENCES | EFFICACY | VIVO | TRANSDUCTION | MODEL | DATABASE | BIOTECHNOLOGY & APPLIED MICROBIOLOGY | HEMOPHILIA-B | FACTOR-IX | GENETICS & HEREDITY | EXPRESSION | VECTORS | 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 | Original
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7. Full Text Transgene regulation using the tetracycline-inducible TetR-KRAB system after AAV-mediated gene transfer in rodents and nonhuman primates
by Le Guiner, Caroline and Stieger, Knut and Toromanoff, Alice and Guilbaud, Mickaël and Mendes-Madeira, Alexandra and Devaux, Marie and Guigand, Lydie and Cherel, Yan and Moullier, Philippe and Rolling, Fabienne and Adjali, Oumeya
PLoS ONE, ISSN 1932-6203, 09/2014, Volume 9, Issue 9, pp. e102538 - e102538
Numerous studies have demonstrated the efficacy of the Adeno-Associated Virus (AAV)-based gene delivery platform in vivo. The control of transgene expression... 
SKELETAL-MUSCLE | VIRAL VECTOR | RECOMBINANT-ADENOASSOCIATED-VIRUS | TIGHT CONTROL | INTRAMUSCULAR INJECTION | IMMUNE-RESPONSES | MULTIDISCIPLINARY SCIENCES | FACTOR-IX | IN-VIVO | MAMMALIAN-CELLS | EXPRESSION | Dependovirus - genetics | Retina - metabolism | Genetic Vectors - administration & dosage | Rats, Wistar | Humans | Tetracycline - metabolism | Male | Muscle, Skeletal - metabolism | Muscle, Skeletal - immunology | Kruppel-Like Transcription Factors - metabolism | Macaca | Muscle, Skeletal - virology | Transgenes | Gene Transfer Techniques | Mice, Inbred C57BL | Tetracycline - pharmacology | Rats | Dependovirus - immunology | Dependovirus - classification | Retina - virology | Gene Expression Regulation - drug effects | Genetic Vectors - drug effects | Animals | Doxycycline | Mice | Immunity, Cellular | Kruppel-Like Transcription Factors - genetics | Proteins | Technology application | Rodents | Genes | Muscles | Primates | Gene expression | Health aspects | Regulations | Animal models | Gene regulation | Viruses | Immunity | Unmanned aerial vehicles | Deoxyribonucleic acid--DNA | Recombinant | Immune system | Cytomegalovirus | Translation | Immune response | Gene transfer | Therapeutic applications | Rapamycin | Vectors | Mammals | Monkeys & apes | Skeletal muscle | Immune systems | Musculoskeletal system | Gene therapy | Regulatory proteins | Index Medicus | Life Sciences | Human health and pathology | Deoxyribonucleic acid | DNA
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8. Full Text Vascular Delivery of Allogeneic MuStem Cells in Dystrophic Dogs Requires Only Short-Term Immunosuppression to Avoid Host Immunity and Generate Clinical/Tissue Benefits
by Lorant, Judith and Larcher, Thibaut and Jaulin, Nicolas and Hedan, Benoît and Lardenois, Aurélie and Leroux, Isabelle and Dubreil, Laurence and Ledevin, Mireille and Goubin, Hélicia and Moullec, Sophie and Deschamps, Jack-Yves and Thorin, Chantal and André, Catherine and Adjali, Oumeya and Rouger, Karl
Cell Transplantation, ISSN 0963-6897, 7/2018, Volume 27, Issue 7, pp. 1096 - 1110
Growing demonstrations of regenerative potential for some stem cells led recently to promising therapeutic proposals for neuromuscular diseases. We have shown... 
immunosuppression | GRMD dog | cell therapy | Duchenne muscular dystrophy | Stem cells | transplantation | MEDICINE, RESEARCH & EXPERIMENTAL | H9C2 CARDIOMYOBLAST CELLS | SYSTEMIC DELIVERY | SATELLITE CELLS | RENAL-TRANSPLANTATION | MESENCHYMAL STEM-CELLS | CELL & TISSUE ENGINEERING | CYCLOSPORINE-A | DUCHENNE MUSCULAR-DYSTROPHY | SKELETAL-MUSCLE REGENERATION | STROMAL CELLS | MYOBLAST TRANSPLANTATION | Life Sciences | Genetics | Original
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9. Full Text 5 years of successful inducible transgene expression following locoregional AAV delivery in nonhuman primates with no detectable immunity
by Guilbaud, Mickaël and Devaux, Marie and Couzinié, Célia 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, 02/2019
Life Sciences | Human health and pathology
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10. Full Text 5 years of successful inducible transgene expression following locoregional AAV delivery in nonhuman primates with no detectable immunity
by Guilbaud, Mickaël and Devaux, Marie and Couzinié, Célia and Le Duff, Johanne and Toromanoff, Alice and Vandamme, Celine 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, 02/2019
Anti-transgene immune responses elicited after intramuscular (IM) delivery of recombinant Adeno-Associated Viruses (rAAV) have been shown to hamper long-term... 
Life Sciences
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11. Full Text Lack of Immunotoxicity After Regional Intravenous (RI) Delivery of rAAV to Nonhuman Primate Skeletal Muscle
by Toromanoff, Alice and Adjali, Oumeya and Larcher, Thibaut and Hill, Marcelo and Guigand, Lydie and Chenuaud, Pierre and Deschamps, Jack-Yves and Gauthier, Olivier and Blancho, Gilles and Vanhove, Bernard and Rolling, Fabienne and Chérel, Yan and Moullier, Philippe and Anegon, Ignacio and Le Guiner, Caroline
Molecular Therapy, ISSN 1525-0016, 01/2010, Volume 18, Issue 1, pp. 151 - 160
In the absence of an immune response from the host, intramuscular (IM) injection of recombinant adeno-associated virus (rAAV) results in the permanent... 
MEDICINE, RESEARCH & EXPERIMENTAL | IMMUNE-RESPONSE | LONG-TERM CORRECTION | REGULATED TRANSGENE EXPRESSION | CANINE HEMOPHILIA-B | MEDIATED GENE-TRANSFER | T-CELL | COAGULATION FACTOR-IX | RECOMBINANT-ADENOASSOCIATED-VIRUS | LIPOPROTEIN-LIPASE DEFICIENCY | BIOTECHNOLOGY & APPLIED MICROBIOLOGY | INTRAMUSCULAR INJECTION | GENETICS & HEREDITY | Immunohistochemistry | Dependovirus - genetics | Enzyme-Linked Immunosorbent Assay | Genetic Vectors - administration & dosage | Genetic Vectors - immunology | Dependovirus - immunology | Injections, Intravenous - adverse effects | Male | Muscle, Skeletal - metabolism | Muscle Fibers, Skeletal - drug effects | Reverse Transcriptase Polymerase Chain Reaction | Genetic Vectors - genetics | Injections, Intramuscular - adverse effects | Animals | In Situ Hybridization | Muscle, Skeletal - drug effects | Muscle Fibers, Skeletal - pathology | Macaca | Transduction, Genetic - methods | Index Medicus | Original
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12. Full Text Accurate Titration of Infectious AAV Particles Requires Measurement of Biologically Active Vector Genomes and Suitable Controls
by François, Achille and Bouzelha, Mohammed and Lecomte, Emilie and Broucque, Frédéric and Penaud-Budloo, Magalie and Adjali, Oumeya and Moullier, Philippe and Blouin, Véronique and Ayuso, Eduard
Molecular Therapy - Methods & Clinical Development, ISSN 2329-0501, 09/2018, Volume 10, pp. 223 - 236
Although the clinical use of recombinant adeno-associated virus (rAAV) vectors is constantly increasing, the development of suitable quality control methods is... 
gene therapy | AAV vectors | quality control | titration | infectivity | ADENOASSOCIATED VIRUS TYPE-2 | MEDICINE, RESEARCH & EXPERIMENTAL | CAPSID PROTEIN | TRAFFICKING | 2ND-STRAND SYNTHESIS | GENERATION | CELL-LINES | REP | VIRAL VECTORS | ADENOVIRUS | TITER | Life Sciences | Biotechnology | Pharmaceutical sciences
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13. Full Text 697. Phenotypic and Functional Characterisation of Human Anti-AAV CD8+ T Cells Using MHC Class I Tetramer-Associated Magnetic Enrichment
by Vandamme, Celine and Devaux, Marie and Jaulin, Nicolas and Guilbaud, Mickaël and Duff, Johanne Le and Hesnard, Leslie and Djamai, Hanane and Bonneville, Marc and Moullier, Philippe and Saulquin, Xavier and Adjali, Oumeya
Molecular Therapy, ISSN 1525-0016, 05/2016, Volume 24, pp. S275 - S276
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14. Full Text Lentiviral transduction of immune cells
by Swainson, Louise and Mongellaz, Cedric and Adjali, Oumeya and Vicente, Rita and Taylor, Naomi
Methods in molecular biology (Clifton, N.J.), ISSN 1064-3745, 2008, Volume 415, pp. 301 - 320
Gene transfer into mammalian cells has been of crucial importance for studies determining the role of specific genes in the differentiation and cell fate of... 
Centrifugation | Cells, Cultured | Virion - metabolism | Leukocytes, Mononuclear - virology | Promoter Regions, Genetic - genetics | Genetic Vectors - genetics | T-Lymphocytes - virology | Animals | Transfection | Plasmids - genetics | B-Lymphocytes - virology | Titrimetry | Lentivirus - genetics | Mice | Dendritic Cells - virology | Hematopoietic Stem Cells - virology | Transgenes | Transduction, Genetic - methods | Index Medicus | Promoter Regions, Genetic | Transduction, Genetic | Dendritic Cells | T-Lymphocytes | Biochemistry, Molecular Biology | B-Lymphocytes | Leukocytes, Mononuclear | Lentivirus | Life Sciences | Virion | Plasmids | Genetic Vectors | Hematopoietic Stem Cells
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