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Dual AAV Gene Therapy for Duchenne Muscular Dystrophy with a 7-kb Mini-Dystrophin Gene in the Canine Model

Dual adeno-associated virus (AAV) technology was developed in 2000 to double the packaging capacity of the AAV vector. The proof of principle has been demonstrated in various mouse models. Yet, pivotal evidence is lacking in large animal models of human diseases. Here we report expression of a 7-kb...

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Detalhes bibliográficos
Publicado no:Hum Gene Ther
Main Authors: Kodippili, Kasun, Hakim, Chady H., Pan, Xiufang, Yang, Hsiao T., Yue, Yongping, Zhang, Yadong, Shin, Jin-Hong, Yang, N. Nora, Duan, Dongsheng
Formato: Artigo
Idioma:Inglês
Publicado em: Mary Ann Liebert, Inc. 2018
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Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC5865264/
https://ncbi.nlm.nih.gov/pubmed/28793798
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/hum.2017.095
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