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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Detaylı Bibliyografya
Yayımlandı:Hum Gene Ther
Asıl Yazarlar: Kodippili, Kasun, Hakim, Chady H., Pan, Xiufang, Yang, Hsiao T., Yue, Yongping, Zhang, Yadong, Shin, Jin-Hong, Yang, N. Nora, Duan, Dongsheng
Materyal Türü: Artigo
Dil:Inglês
Baskı/Yayın Bilgisi: Mary Ann Liebert, Inc. 2018
Konular:
Research Articles
Online Erişim: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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