Base editing rescue of spinal muscular atrophy in cells and in mice
Mandana Arbab(Broad Institute), David R. Liu(Broad Institute), Guangping Gao(University of Massachusetts Chan Medical School), W. David Arnold(University of Missouri Health System), Żaneta Matuszek(Broad Institute), Jun Xie(Hainan University), Jonathan M. Levy(Broad Institute), Arthur H.M. Burghes(University of Toronto), Gregory A. Newby(Broad Institute), Kevin T. Zhao(Broad Institute), Max W. Shen(Beth Israel Deaconess Medical Center), Ailing Du(University of Massachusetts Chan Medical School), Anton J. Blatnik(The Ohio State University Wexner Medical Center), Kaitlyn M. Kray(The Ohio State University Wexner Medical Center), Aditya Raguram(Broad Institute), Dan Wang(Chinese People's Armed Police Force Medical College Affiliated Hospital), Michelle F. Richter(Broad Institute)
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