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C-Terminal-Truncated Microdystrophin Recruits Dystrobrevin and Syntrophin to the Dystrophin-Associated Glycoprotein Complex and Reduces Muscular Dystrophy in Symptomatic Utrophin/Dystrophin Double-Knockout Mice

C-terminal-truncated (ΔC) microdystrophin is being developed for Duchenne muscular dystrophy gene therapy. Encouraging results have been achieved in the mdx mouse model. Unfortunately, mdx mice do not display the same phenotype as human patients. Evaluating ΔC microdystrophin in a symptomatic model...

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Bibliografski detalji
Glavni autori: Yue, Yongping, Liu, Mingju, Duan, Dongsheng
Format: Artigo
Jezik:Inglês
Izdano: 2006
Teme:
Online pristup:https://ncbi.nlm.nih.gov/pmc/articles/PMC2581714/
https://ncbi.nlm.nih.gov/pubmed/16563874
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymthe.2006.01.007
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