Article
CRISPR/Cas9 editing of directly reprogrammed myogenic progenitors restores dystrophin expression in a mouse model of muscular dystrophy.
Stem cell reports - 8 Feb 2022
Domenig Seraina A, Bundschuh Nicola, Lenardič Ajda, Ghosh Adhideb, Kim Inseon, Qabrati Xhem, D'Hulst Gommaar, Bar-Nur Ori
Abstract excerpt
Genetic mutations in dystrophin manifest in Duchenne muscular dystrophy (DMD), the most commonly inherited muscle disease. Here, we report on reprogramming of fibroblasts from two DMD mouse models into induced myogenic progenitor cells (iMPCs) by MyoD overexpression in concert with small molecule treatment. DMD iMPCs proliferate extensively, while expressing myogenic stem cell markers including Pax7 and Myf5....
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