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Page 1
Long-term microdystrophin gene therapy is effective in a canine model of Duchenne muscular dystrophy.
Le Guiner C, Servais L, Montus M, Larcher T, Fraysse B, Moullec S, Allais M, François V, Dutilleul M, Malerba A, Koo T, Thibaut JL, Matot B, Devaux M, Le Duff J, Deschamps JY, Barthelemy I, Blot S, Testault I, Wahbi K, Ederhy S, Martin S, Veron P, Georger C, Athanasopoulos T, Masurier C, Mingozzi F, Carlier P, Gjata B, Hogrel JY, Adjali O, Mavilio F, Voit T, Moullier P, Dickson G. Le Guiner C, et al. Among authors: le duff j. Nat Commun. 2017 Jul 25;8:16105. doi: 10.1038/ncomms16105. Nat Commun. 2017. PMID: 28742067 Free PMC article.
Forelimb treatment in a large cohort of dystrophic dogs supports delivery of a recombinant AAV for exon skipping in Duchenne patients.
Le Guiner C, Montus M, Servais L, Cherel Y, Francois V, Thibaud JL, Wary C, Matot B, Larcher T, Guigand L, Dutilleul M, Domenger C, Allais M, Beuvin M, Moraux A, Le Duff J, Devaux M, Jaulin N, Guilbaud M, Latournerie V, Veron P, Boutin S, Leborgne C, Desgue D, Deschamps JY, Moullec S, Fromes Y, Vulin A, Smith RH, Laroudie N, Barnay-Toutain F, Rivière C, Bucher S, Le TH, Delaunay N, Gasmi M, Kotin RM, Bonne G, Adjali O, Masurier C, Hogrel JY, Carlier P, Moullier P, Voit T. Le Guiner C, et al. Among authors: le duff j. Mol Ther. 2014 Nov;22(11):1923-35. doi: 10.1038/mt.2014.151. Epub 2014 Aug 4. Mol Ther. 2014. PMID: 25200009 Free PMC article.
Generation and in vivo evaluation of IL10-treated dendritic cells in a nonhuman primate model of AAV-based gene transfer.
Moreau A, Vandamme C, Segovia M, Devaux M, Guilbaud M, Tilly G, Jaulin N, Le Duff J, Cherel Y, Deschamps JY, Anegon I, Moullier P, Cuturi MC, Adjali O. Moreau A, et al. Among authors: le duff j. Mol Ther Methods Clin Dev. 2014 Jul 23;1:14028. doi: 10.1038/mtm.2014.28. eCollection 2014. Mol Ther Methods Clin Dev. 2014. PMID: 26015970 Free PMC article.
Five Years of Successful Inducible Transgene Expression Following Locoregional Adeno-Associated Virus Delivery in Nonhuman Primates with No Detectable Immunity.
Guilbaud M, Devaux M, Couzinié C, Le Duff J, Toromanoff A, Vandamme C, Jaulin N, Gernoux G, Larcher T, Moullier P, Le Guiner C, Adjali O. Guilbaud M, et al. Among authors: le duff j. Hum Gene Ther. 2019 Jul;30(7):802-813. doi: 10.1089/hum.2018.234. Epub 2019 Apr 16. Hum Gene Ther. 2019. PMID: 30808235 Free PMC article.
AAV8 locoregional delivery induces long-term expression of an immunogenic transgene in macaques despite persisting local inflammation.
Gernoux G, Guilbaud M, Devaux M, Journou M, Pichard V, Jaulin N, Léger A, Le Duff J, Deschamps JY, Le Guiner C, Moullier P, Cherel Y, Adjali O. Gernoux G, et al. Among authors: le duff j. Mol Ther Methods Clin Dev. 2021 Feb 6;20:660-674. doi: 10.1016/j.omtm.2021.02.003. eCollection 2021 Mar 12. Mol Ther Methods Clin Dev. 2021. PMID: 33718516 Free PMC article.