Intravenous Administration of a MTMR2-Encoding AAV Vector Ameliorates the Phenotype of Myotubular Myopathy in Mice

X-linked myotubular myopathy (XLMTM) is a severe congenital disorder in male infants that leads to generalized skeletal muscle weakness and is frequently associated with fatal respiratory failure. XLMTM is caused by loss-of-function mutations in the MTM1 gene, which encodes myotubularin, the founder...

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Publicat a:J Neuropathol Exp Neurol
Autors principals: Danièle, Nathalie, Moal, Christelle, Julien, Laura, Marinello, Martina, Jamet, Thibaud, Martin, Samia, Vignaud, Alban, Lawlor, Michael W, Buj-Bello, Ana
Format: Artigo
Idioma:Inglês
Publicat: Oxford University Press 2018
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Original Articles
Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC5939852/
https://ncbi.nlm.nih.gov/pubmed/29408998
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1093/jnen/nly002
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