In vivo genome editing restores dystrophin expression and cardiac function in dystrophic mice

Mona El Refaey, Li Xu, Yandi Gao, Benjamin D. Canan, T. M. Ayodele Adesanya, Sarah C. Warner, Keiko Akagi, David E. Symer, Peter J. Mohler, Jianjie Ma, Paul M.L. Janssen, Renzhi Han

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108 Scopus citations

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Medicine & Life Sciences