Single-cut genome editing restores dystrophin expression in a new mouse model of muscular dystrophy

Leonela Amoasii, Chengzu Long, Hui Li, Alex A. Mireault, John M. Shelton, Efrain Sanchez-Ortiz, John R. McAnally, Samadrita Bhattacharyya, Florian Schmidt, Dirk Grimm, Stephen D. Hauschka, Rhonda Bassel-Duby, Eric N. Olson

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174 Scopus citations

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Medicine & Life Sciences