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Safe and bodywide muscle transduction in young adult Duchenne muscular dystrophy dogs with adeno-associated virus

The ultimate goal of muscular dystrophy gene therapy is to treat all muscles in the body. Global gene delivery was demonstrated in dystrophic mice more than a decade ago using adeno-associated virus (AAV). However, translation to affected large mammals has been challenging. The only reported attempt...

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Dettagli Bibliografici
Pubblicato in:Hum Mol Genet
Autori principali: Yue, Yongping, Pan, Xiufang, Hakim, Chady H., Kodippili, Kasun, Zhang, Keqing, Shin, Jin-Hong, Yang, Hsiao T., McDonald, Thomas, Duan, Dongsheng
Natura: Artigo
Lingua:Inglês
Pubblicazione: Oxford University Press 2015
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Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC4581611/
https://ncbi.nlm.nih.gov/pubmed/26264580
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1093/hmg/ddv310
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