Gene Therapy Prolongs Survival and Restores Function in Murine and Canine Models of Myotubular Myopathy

Loss-of-function mutations in the myotubularin gene (MTM1) cause X-linked myotubular myopathy (XLMTM), a fatal, congenital pediatric disease that affects the entire skeletal musculature. Systemic administration of a single dose of a recombinant serotype-8 adeno-associated virus (AAV8) vector express...

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Main Authors: Childers, Martin K, Joubert, Romain, Poulard, Karine, Moal, Christelle, Grange, Robert W, Doering, Jonathan A, Lawlor, Michael W, Rider, Branden E., Jamet, Thibaud, Danièle, Nathalie, Martin, Samia, Rivière, Christel, Soker, Thomas, Hammer, Caroline, Van Wittenberghe, Laetitia, Lockard, Mandy, Guan, Xuan, Goddard, Melissa, Mitchell, Erin, Barber, Jane, Williams, J. Koudy, Mack, David L, Furth, Mark E, Vignaud, Alban, Masurier, Carole, Mavilio, Fulvio, Moullier, Philippe, Beggs, Alan H, Buj-Bello, Anna
Format: Artigo
Language:Inglês
Published: 2014
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Online Access:https://ncbi.nlm.nih.gov/pmc/articles/PMC4105197/
https://ncbi.nlm.nih.gov/pubmed/24452262
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1126/scitranslmed.3007523
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