Systemic gene transfer reveals distinctive muscle transduction profile of tyrosine mutant AAV-1, -6, and -9 in neonatal dogs

The muscular dystrophies are a group of devastating genetic disorders that affect both skeletal and cardiac muscle. An effective gene therapy for these diseases requires bodywide muscle delivery. Tyrosine mutant adeno-associated virus (AAV) has been considered as a class of highly potent gene transf...

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Main Authors: Hakim, Chady H, Yue, Yongping, Shin, Jin-Hong, Williams, Regina R, Zhang, Keqing, Smith, Bruce F, Duan, Dongsheng
Format: Artigo
Jezik:Inglês
Izdano: Nature Publishing Group 2014
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Article
Online dostop:https://ncbi.nlm.nih.gov/pmc/articles/PMC4121663/
https://ncbi.nlm.nih.gov/pubmed/25105153
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mtm.2014.2
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