Systemic Trans-Splicing Adeno-Associated Viral Delivery Efficiently Transduces the Heart of Adult mdx Mouse, a Model for Duchenne Muscular Dystrophy

Trans-splicing adeno-associated viral (tsAAV) vectors hold great promise for delivering large therapeutic genes. One potential application is in the treatment of Duchenne muscular dystrophy (DMD). In this case, it is necessary to transduce whole body muscle. We demonstrated body-wide AAV-9 tsAAV tra...

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Bibliografiska uppgifter
Huvudupphovsmän: Ghosh, Arkasubhra, Yue, Yongping, Shin, Jin-Hong, Duan, Dongsheng
Materialtyp: Artigo
Språk:Inglês
Publicerad: Mary Ann Liebert, Inc. 2009
Ämnen:
Scientific Articles
Länkar:https://ncbi.nlm.nih.gov/pmc/articles/PMC2829465/
https://ncbi.nlm.nih.gov/pubmed/19627234
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/hum.2009.058
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