Therapeutic Exon Skipping via a CRISPR-guided Cytidine Deaminase Rescues Dystrophic Cardiomyopathy In Vivo

Jia Li, Kaiying Wang, Yuchen Zhang,Tuan Qi,Juanjuan Yuan, Lei Zhang, Han Qiu,Jinxi Wang,Huang-Tian Yang,Yi Dai,Yan Song,Xing Chang

Circulation（2021）

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Background: Loss of dystrophin protein causes Duchenne muscular dystrophy (DMD), characterized by progressive degeneration of cardiac and skeletal muscles, and mortality in adolescence or young adu...

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