Enhanced CRISPR-Cas9 correction of Duchenne muscular dystrophy in mice by a self-complementary AAV delivery system

Yu Zhang, Hui Li, Yi Li Min, Efrain Sanchez-Ortiz, Jian Huang, Alex A. Mireault, John M. Shelton, Jiwoong Kim, Pradeep P.A. Mammen, Rhonda Bassel-Duby, Eric N. Olson

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108 Scopus citations

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Medicine & Life Sciences