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Adeno-Associated Virus-Mediated Microdystrophin Expression Protects Young mdx Muscle from Contraction-Induced Injury

Duchenne muscular dystrophy (DMD) is the most common inherited lethal muscle degenerative disease. Currently there is no cure. Highly abbreviated microdystrophin cDNAs were developed recently for adeno-associated virus (AAV)-mediated DMD gene therapy. Among these, a C-terminal-truncated ΔR4-R23/ΔC m...

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Autors principals: Liu, Mingju, Yue, Yongping, Harper, Scott Q., Grange, Robert W., Chamberlain, Jeffrey S., Duan, Dongsheng
Format: Artigo
Idioma:Inglês
Publicat: 2005
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Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC2581717/
https://ncbi.nlm.nih.gov/pubmed/15668136
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymthe.2004.09.013
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