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100-fold but not 50-fold dystrophin overexpression aggravates electrocardiographic defects in the mdx model of Duchenne muscular dystrophy

Dystrophin gene replacement holds the promise of treating Duchenne muscular dystrophy. Supraphysiological expression is a concern for all gene therapy studies. In the case of Duchenne muscular dystrophy, Chamberlain and colleagues found that 50-fold overexpression did not cause deleterious side effe...

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Bibliografiske detaljer
Udgivet i:Mol Ther Methods Clin Dev
Main Authors: Yue, Yongping, Wasala, Nalinda B, Bostick, Brian, Duan, Dongsheng
Format: Artigo
Sprog:Inglês
Udgivet: Nature Publishing Group 2016
Fag:
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC4934459/
https://ncbi.nlm.nih.gov/pubmed/27419194
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mtm.2016.45
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