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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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Bibliografski detalji
Izdano u:Hum Gene Ther
Glavni autori: Kodippili, Kasun, Hakim, Chady H., Pan, Xiufang, Yang, Hsiao T., Yue, Yongping, Zhang, Yadong, Shin, Jin-Hong, Yang, N. Nora, Duan, Dongsheng
Format: Artigo
Jezik:Inglês
Izdano: Mary Ann Liebert, Inc. 2018
Teme:
Online pristup: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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