Long-term effects of systemic gene therapy in a canine model of myotubular myopathy

INTRODUCTION: X-linked myotubular myopathy (XLMTM), a devastating pediatric disease caused by the absence of the protein myotubularin, results from mutations in the MTM1 gene. While there is no cure for XLMTM, we previously reported effects of MTM1 gene therapy using adeno-associated viral (AAV) vec...

Fuld beskrivelse

Na minha lista:
Bibliografiske detaljer
Udgivet i:Muscle Nerve
Main Authors: Elverman, Matthew, Goddard, Melissa A, Mack, David, Snyder, Jessica M, Lawlor, Michael W, Meng, Hui, Beggs, Alan H, Buj-Bello, Ana, Poulard, Karine, Marsh, Anthony P, Grange, Robert W, Kelly, Valerie E., Childers, Martin K
Format: Artigo
Sprog:Inglês
Udgivet: 2017
Fag:
Article
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC5620115/
https://ncbi.nlm.nih.gov/pubmed/28370029
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1002/mus.25658
Tags: Tilføj Tag
Ingen Tags, Vær først til at tagge denne postø!

Lignende værker