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Systemic AAV8-Mediated Gene Therapy Drives Whole-Body Correction of Myotubular Myopathy in Dogs.
Mack DL, Poulard K, Goddard MA, Latournerie V, Snyder JM, Grange RW, Elverman MR, Denard J, Veron P, Buscara L, Le Bec C, Hogrel JY, Brezovec AG, Meng H, Yang L, Liu F, O'Callaghan M, Gopal N, Kelly VE, Smith BK, Strande JL, Mavilio F, Beggs AH, Mingozzi F, Lawlor MW, Buj-Bello A, Childers MK. Mack DL, et al. Among authors: mavilio f. Mol Ther. 2017 Apr 5;25(4):839-854. doi: 10.1016/j.ymthe.2017.02.004. Epub 2017 Feb 22. Mol Ther. 2017. PMID: 28237839 Free PMC article.
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: mavilio f. Sci Transl Med. 2014 Jan 22;6(220):220ra10. doi: 10.1126/scitranslmed.3007523. Sci Transl Med. 2014. PMID: 24452262 Free PMC article.
Cerebellar Pathology in an Inducible Mouse Model of Friedreich Ataxia.
Mercado-Ayón E, Warren N, Halawani S, Rodden LN, Ngaba L, Dong YN, Chang JC, Fonck C, Mavilio F, Lynch DR, Lin H. Mercado-Ayón E, et al. Among authors: mavilio f. Front Neurosci. 2022 Mar 24;16:819569. doi: 10.3389/fnins.2022.819569. eCollection 2022. Front Neurosci. 2022. PMID: 35401081 Free PMC article.
Gene Therapy for Hemoglobinopathies.
Cavazzana M, Mavilio F. Cavazzana M, et al. Among authors: mavilio f. Hum Gene Ther. 2018 Oct;29(10):1106-1113. doi: 10.1089/hum.2018.122. Hum Gene Ther. 2018. PMID: 30200783 Free PMC article. Review.
Gene therapy: back on track?
Mavilio F. Mavilio F. EMBO Rep. 2010 Feb;11(2):75. doi: 10.1038/embor.2009.284. EMBO Rep. 2010. PMID: 20118988 Free PMC article. No abstract available.
Correction of beta-thalassemia major by gene transfer in haematopoietic progenitors of pediatric patients.
Roselli EA, Mezzadra R, Frittoli MC, Maruggi G, Biral E, Mavilio F, Mastropietro F, Amato A, Tonon G, Refaldi C, Cappellini MD, Andreani M, Lucarelli G, Roncarolo MG, Marktel S, Ferrari G. Roselli EA, et al. Among authors: mavilio f. EMBO Mol Med. 2010 Aug;2(8):315-28. doi: 10.1002/emmm.201000083. EMBO Mol Med. 2010. PMID: 20665635 Free PMC article.
A single epidermal stem cell strategy for safe ex vivo gene therapy.
Droz-Georget Lathion S, Rochat A, Knott G, Recchia A, Martinet D, Benmohammed S, Grasset N, Zaffalon A, Besuchet Schmutz N, Savioz-Dayer E, Beckmann JS, Rougemont J, Mavilio F, Barrandon Y. Droz-Georget Lathion S, et al. Among authors: mavilio f. EMBO Mol Med. 2015 Apr;7(4):380-93. doi: 10.15252/emmm.201404353. EMBO Mol Med. 2015. PMID: 25724200 Free PMC article.
196 results