Single Intramuscular Injection of AAV-shRNA Reduces DNM2 and Prevents Myotubular Myopathy in Mice

Myotubular myopathy, or X-linked centronuclear myopathy, is a severe muscle disorder representing a significant burden for patients and their families. It is clinically characterized by neonatal and severe muscle weakness and atrophy. Mutations in the myotubularin (MTM1) gene cause myotubular myopat...

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Bibliografski detalji
Izdano u:Mol Ther
Glavni autori: Tasfaout, Hichem, Lionello, Valentina M., Kretz, Christine, Koebel, Pascale, Messaddeq, Nadia, Bitz, Deborah, Laporte, Jocelyn, Cowling, Belinda S.
Format: Artigo
Jezik:Inglês
Izdano: American Society of Gene & Cell Therapy 2018
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Original Article
Online pristup:https://ncbi.nlm.nih.gov/pmc/articles/PMC6080128/
https://ncbi.nlm.nih.gov/pubmed/29506908
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymthe.2018.02.008
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