ArticleHuman gene therapy2011

Delivery of AAV2/9-microdystrophin genes incorporating helix 1 of the coiled-coil motif in the C-terminal domain of dystrophin improves muscle pathology and restores the level of α1-syntrophin and α-dystrobrevin in skeletal muscles of mdx mice.

Taeyoung Koo et al.PubMed ↗Full text ↗Publisher ↗

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