Structure-Based Designed Nano-Dysferlin Significantly Improves Dysferlinopathy in BLA/J Mice

Dysferlinopathy is an autosomal recessive muscular dystrophy characterized by the progressive loss of motility that is caused by mutations throughout the DYSF gene. There are currently no approved therapies that ameliorate or reverse dysferlinopathy. Gene delivery using adeno-associated vectors (AAV...

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Bibliografiske detaljer
Udgivet i:Mol Ther
Main Authors: Llanga, Telmo, Nagy, Nadia, Conatser, Laura, Dial, Catherine, Sutton, R. Bryan, Hirsch, Matthew L.
Format: Artigo
Sprog:Inglês
Udgivet: American Society of Gene & Cell Therapy 2017
Fag:
Original Article
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC5589059/
https://ncbi.nlm.nih.gov/pubmed/28629822
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymthe.2017.05.013
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