Phase 1 Gene Therapy for Duchenne Muscular Dystrophy Using a Translational Optimized AAV Vector

Efficient and widespread gene transfer is required for successful treatment of Duchenne muscular dystrophy (DMD). Here, we performed the first clinical trial using a chimeric adeno-associated virus (AAV) capsid variant (designated AAV2.5) derived from a rational design strategy. AAV2.5 was generated...

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Detalhes bibliográficos
Main Authors: Bowles, Dawn E, McPhee, Scott WJ, Li, Chengwen, Gray, Steven J, Samulski, Jade J, Camp, Angelique S, Li, Juan, Wang, Bing, Monahan, Paul E, Rabinowitz, Joseph E, Grieger, Joshua C, Govindasamy, Lakshmanan, Agbandje-McKenna, Mavis, Xiao, Xiao, Samulski, R Jude
Formato: Artigo
Idioma:Inglês
Publicado em: Nature Publishing Group 2012
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Original Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC3277234/
https://ncbi.nlm.nih.gov/pubmed/22068425
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2011.237
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