Improvement of the mdx mouse dystrophic phenotype by systemic in utero AAV8 delivery of a minidystrophin gene

Duchenne muscular dystrophy (DMD) is a devastating primary muscle disease with pathological changes in skeletal muscle that are ongoing at birth. Progressive deterioration in striated muscle function in affected individuals ultimately results in early death due to cardio-pulmonary failure. Since aff...

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Detalhes bibliográficos
Main Authors: Koppanati, Bhanu Munil, Li, Juan, Reay, Dan P., Wang, Bing, Daood, Molly, Zheng, Heng, Xiao, Xiao, Watchko, Jon F., Clemens, Paula R.
Formato: Artigo
Idioma:Inglês
Publicado em: 2010
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Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC2939256/
https://ncbi.nlm.nih.gov/pubmed/20535217
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/gt.2010.84
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