Somatic gene editing ameliorates skeletal and cardiac muscle failure in pig and human models of Duchenne muscular dystrophy.
Moretti A, Fonteyne L, Giesert F, Hoppmann P, Meier AB, Bozoglu T, Baehr A, Schneider CM, Sinnecker D, Klett K, Fröhlich T, Rahman FA, Haufe T, Sun S, Jurisch V, Kessler B, Hinkel R, Dirschinger R, Martens E, Jilek C, Graf A, Krebs S, Santamaria G, Kurome M, Zakhartchenko V, Campbell B, Voelse K, Wolf A, Ziegler T, Reichert S, Lee S, Flenkenthaler F, Dorn T, Jeremias I, Blum H, Dendorfer A, Schnieke A, Krause S, Walter MC, Klymiuk N, Laugwitz KL, Wolf E, Wurst W, Kupatt C.
Moretti A, et al.
Nat Med. 2020 Feb;26(2):207-214. doi: 10.1038/s41591-019-0738-2. Epub 2020 Jan 27.
Nat Med. 2020.
PMID: 31988462
Free PMC article.