Micro-utrophin Improves Cardiac and Skeletal Muscle Function of Severely Affected D2/mdx Mice

Duchenne muscular dystrophy (DMD) is an X-linked muscle-wasting disease caused by mutations in the dystrophin gene. DMD boys are wheelchair-bound around 12 years and generally survive into their twenties. There is currently no effective treatment except palliative care, although personalized treatme...

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Publicat a:Mol Ther Methods Clin Dev
Autors principals: Kennedy, Tahnee L., Guiraud, Simon, Edwards, Ben, Squire, Sarah, Moir, Lee, Babbs, Arran, Odom, Guy, Golebiowski, Diane, Schneider, Joel, Chamberlain, Jeffrey S., Davies, Kay E.
Format: Artigo
Idioma:Inglês
Publicat: American Society of Gene & Cell Therapy 2018
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Article
Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC6216100/
https://ncbi.nlm.nih.gov/pubmed/30417024
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtm.2018.10.005
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