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CRISPR/Cas9 editing of directly reprogrammed myogenic progenitors restores dystrophin expression in a mouse model of muscular dystrophy

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 tr...

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Detalles Bibliográficos
Autores principales:	Domenig, Seraina A., Bundschuh, Nicola, Lenardič, Ajda, Ghosh, Adhideb, Kim, Inseon, Qabrati, Xhem, D'Hulst, Gommaar, Bar-Nur, Ori
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Elsevier 2022
Materias:	Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8828535/ https://www.ncbi.nlm.nih.gov/pubmed/34995499 http://dx.doi.org/10.1016/j.stemcr.2021.12.003

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