Activation of endogenous full-length utrophin by MyoAAV-UA as a therapeutic approach for Duchenne muscular dystrophy

Activation of endogenous full-length utrophin by MyoAAV-UA as a therapeutic approach for Duchenne muscular dystrophy

Abstract Activation of endogenous full-length utrophin, a dystrophin homolog, presents an attractive therapeutic strategy for Duchenne muscular dystrophy (DMD), regardless of mutation types and loci. However, current dCas9-based activators are too large for efficient adeno-associated virus delivery,...

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Main Authors: Ruo Wu, Peng Li, Puhao Xiao, Shu Zhang, Xiaopeng Wang, Jie Liu, Wenjie Sun, Yue Chang, Xiuyi Ai, Lijiao Chen, Yan Zhuo, Jiaojian Wang, Zhengbo Wang, Shangang Li, Yuanyuan Li, Weizhi Ji, Wenting Guo, Shiwen Wu, Yongchang Chen
Format: Article
Language:English
Published: Nature Portfolio 2025-03-01
Series:Nature Communications
Online Access:https://doi.org/10.1038/s41467-025-57831-5
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https://doi.org/10.1038/s41467-025-57831-5

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