A humanized knockin mouse model of Duchenne muscular dystrophy and its correction by CRISPR-Cas9 therapeutic gene editing.
Zhang Y, Li H, Nishiyama T, McAnally JR, Sanchez-Ortiz E, Huang J, Mammen PPA, Bassel-Duby R, Olson EN
Mol Ther Nucleic Acids. 2022 Aug 1;29:525-537. doi: 10.1016/j.omtn.2022.07.024. eCollection 2022 Sep 13.
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