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Page 1
Gene therapy prolongs survival and restores function in murine and canine models of myotubular myopathy.
Childers MK, Joubert R, Poulard K, Moal C, Grange RW, Doering JA, Lawlor MW, Rider BE, Jamet T, Danièle N, Martin S, Rivière C, Soker T, Hammer C, Van Wittenberghe L, Lockard M, Guan X, Goddard M, Mitchell E, Barber J, Williams JK, Mack DL, Furth ME, Vignaud A, Masurier C, Mavilio F, Moullier P, Beggs AH, Buj-Bello A. Childers MK, et al. Among authors: moullier p. Sci Transl Med. 2014 Jan 22;6(220):220ra10. doi: 10.1126/scitranslmed.3007523. Sci Transl Med. 2014. PMID: 24452262 Free PMC article.
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: moullier p. Nat Commun. 2017 Jul 25;8:16105. doi: 10.1038/ncomms16105. Nat Commun. 2017. PMID: 28742067 Free PMC article.
Capsid-specific removal of circulating antibodies to adeno-associated virus vectors.
Bertin B, Veron P, Leborgne C, Deschamps JY, Moullec S, Fromes Y, Collaud F, Boutin S, Latournerie V, van Wittenberghe L, Delache B, Le Grand R, Dereuddre-Bosquet N, Benveniste O, Moullier P, Masurier C, Merten O, Mingozzi F. Bertin B, et al. Among authors: moullier p. Sci Rep. 2020 Jan 21;10(1):864. doi: 10.1038/s41598-020-57893-z. Sci Rep. 2020. PMID: 31965041 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: moullier p. 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.
Safe, efficient, and reproducible gene therapy of the brain in the dog models of Sanfilippo and Hurler syndromes.
Ellinwood NM, Ausseil J, Desmaris N, Bigou S, Liu S, Jens JK, Snella EM, Mohammed EE, Thomson CB, Raoul S, Joussemet B, Roux F, Chérel Y, Lajat Y, Piraud M, Benchaouir R, Hermening S, Petry H, Froissart R, Tardieu M, Ciron C, Moullier P, Parkes J, Kline KL, Maire I, Vanier MT, Heard JM, Colle MA. Ellinwood NM, et al. Among authors: moullier p. Mol Ther. 2011 Feb;19(2):251-9. doi: 10.1038/mt.2010.265. Epub 2010 Dec 7. Mol Ther. 2011. PMID: 21139569 Free PMC article.
Short-lived recombinant adeno-associated virus transgene expression in dystrophic muscle is associated with oxidative damage to transgene mRNA.
Dupont JB, Tournaire B, Georger C, Marolleau B, Jeanson-Leh L, Ledevin M, Lindenbaum P, Lecomte E, Cogné B, Dubreil L, Larcher T, Gjata B, Van Wittenberghe L, Le Guiner C, Penaud-Budloo M, Snyder RO, Moullier P, Léger A. Dupont JB, et al. Among authors: moullier p. Mol Ther Methods Clin Dev. 2015 Apr 8;2:15010. doi: 10.1038/mtm.2015.10. eCollection 2015. Mol Ther Methods Clin Dev. 2015. PMID: 26029721 Free PMC article.
146 results