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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Hoofdauteurs: Koppanati, Bhanu Munil, Li, Juan, Reay, Dan P., Wang, Bing, Daood, Molly, Zheng, Heng, Xiao, Xiao, Watchko, Jon F., Clemens, Paula R.
Formaat: Artigo
Taal:Inglês
Gepubliceerd in: 2010
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Online toegang: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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