Micro-dystrophin and follistatin co-delivery restores muscle function in aged DMD model

Pharmacologic strategies have provided modest improvement in the devastating muscle-wasting disease, Duchenne muscular dystrophy (DMD). Pre-clinical gene therapy studies have shown promise in the mdx mouse model; however, studies conducted after disease onset fall short of fully correcting muscle st...

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Autors principals: Rodino-Klapac, Louise R., Janssen, Paul M. L., Shontz, Kimberly M., Canan, Benjamin, Montgomery, Chrystal L., Griffin, Danielle, Heller, Kristin, Schmelzer, Leah, Handy, Chalonda, Clark, K. Reed, Sahenk, Zarife, Mendell, Jerry R., Kaspar, Brian K.
Format: Artigo
Idioma:Inglês
Publicat: Oxford University Press 2013
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Articles
Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC3895965/
https://ncbi.nlm.nih.gov/pubmed/23863459
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1093/hmg/ddt342
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