AAV8 locoregional delivery induces long-term expression of an immunogenic transgene in macaques despite persisting local inflammation

Autor: Gwladys Gernoux, Mickaël Guilbaud, Marie Devaux, Malo Journou, Virginie Pichard, Nicolas Jaulin, Adrien Léger, Johanne Le Duff, Jack-Yves Deschamps, Caroline Le Guiner, Philippe Moullier, Yan Cherel, Oumeya Adjali

Publikováno v: Molecular Therapy: Methods & Clinical Development, Vol 20, Iss , Pp 660-674 (2021)

Adeno-associated virus (AAV) vectors are considered efficient vectors for gene transfer, as illustrated by recent successful clinical trials targeting retinal or neurodegenerative disorders. However, limitations as host immune responses to AAV capsid

Externí odkaz: https://doaj.org/article/38d74ee6093e4b7fa86f324ccc982a8e

Long-term microdystrophin gene therapy is effective in a canine model of Duchenne muscular dystrophy

Autor: Caroline Le Guiner, Laurent Servais, Marie Montus, Thibaut Larcher, Bodvaël Fraysse, Sophie Moullec, Marine Allais, Virginie François, Maeva Dutilleul, Alberto Malerba, Taeyoung Koo, Jean-Laurent Thibaut, Béatrice Matot, Marie Devaux, Johanne Le Duff, Jack-Yves Deschamps, Inès Barthelemy, Stéphane Blot, Isabelle Testault, Karim Wahbi, Stéphane Ederhy, Samia Martin, Philippe Veron, Christophe Georger, Takis Athanasopoulos, Carole Masurier, Federico Mingozzi, Pierre Carlier, Bernard Gjata, Jean-Yves Hogrel, Oumeya Adjali, Fulvio Mavilio, Thomas Voit, Philippe Moullier, George Dickson

Publikováno v: Nature Communications, Vol 8, Iss 1, Pp 1-15 (2017)

Duchenne muscular dystrophy is a progressive degenerative disease of muscles caused by mutations in the dystrophin gene. Here the authors use AAV vectors to deliver microdystrophin to dogs with muscular dystrophy, and show restoration of dystrophin e

Externí odkaz: https://doaj.org/article/d425f7f6bf364aa99c6850ae61ecdd6b

AAV8 locoregional delivery induces long-term expression of an immunogenic transgene in macaques despite persisting local inflammation

Autor: Virginie Pichard, Oumeya Adjali, Nicolas Jaulin, Malo Journou, Johanne Le Duff, Gwladys Gernoux, Adrien Leger, Caroline Le Guiner, Yan Cherel, Marie Devaux, Mickaël Guilbaud, Jack-Yves Deschamps, Philippe Moullier

Publikováno v: Molecular Therapy: Methods & Clinical Development, Vol 20, Iss, Pp 660-674 (2021)
Molecular Therapy-Methods and Clinical Development
Molecular Therapy-Methods and Clinical Development, Nature Publishing Group, 2021, 20, pp.660-674. ⟨10.1016/j.omtm.2021.02.003⟩
Molecular Therapy. Methods & Clinical Development

Adeno-associated virus (AAV) vectors are considered efficient vectors for gene transfer, as illustrated by recent successful clinical trials targeting retinal or neurodegenerative disorders. However, limitations as host immune responses to AAV capsid

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::ddc7495eb50c9a410c7de397d4680321
https://doi.org/10.1016/j.omtm.2021.02.003

5 years of successful inducible transgene expression following locoregional AAV delivery in nonhuman primates with no detectable immunity

Autor: Mickaël Guilbaud, Marie Devaux, Célia Couzinié, Johanne Le Duff, Alice Toromanoff, Céline Vandamme, Nicolas Jaulin, Gwladys Gernoux, Thibaut Larcher, Philippe Moullier, Caroline Le Guiner, Oumeya Adjali

Publikováno v: Human Gene Therapy
Human Gene Therapy, Mary Ann Liebert, 2019, ⟨10.1089/hum.2018.234⟩
Human Gene Therapy, Mary Ann Liebert, 2019
Human Gene Therapy, . (2019)
HAL

5 years of successful inducible transgene expression following locoregional AAV delivery in nonhuman primates with no detectable immunity

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=dedup_wf_001::0c415daec7997b02420d303ae3fac579
https://hal.archives-ouvertes.fr/hal-02091623/document

697. Phenotypic and Functional Characterisation of Human Anti-AAV CD8+ T Cells Using MHC Class I Tetramer-Associated Magnetic Enrichment

Autor: Marc Bonneville, Leslie Hesnard, Philippe Moullier, Celine Vandamme, Johanne Le Duff, Xavier Saulquin, Hanane Djamai, Marie Devaux, Mickaël Guilbaud, Nicolas Jaulin, Oumeya Adjali

Publikováno v: Molecular Therapy. 24:S275-S276

Introduction: Recombinant Adeno-Associated Vectors (AAV) represent nowadays the most largely used platform for in vivo gene therapy. Unfortunately, despite promising results in preclinical and clinical studies, pre-existing immunity against the viral

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_________::65fae802a5a800036b6f93803aa629b3
https://doi.org/10.1016/s1525-0016(16)33505-5