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Effective restoration of dystrophin expression in iPSC (Mdx)-derived muscle progenitor cells using the CRISPR/Cas9 system and homology-directed repair technology

Duchenne muscular dystrophy (DMD) is a progressive myopathic disease caused by mutations in the gene encoding dystrophin protein that eventually leads to the exhaustion of myogenic progenitor cells (MPC). Autologous induced pluripotent stem cells (iPSCs) provide an endless source of MPC, which can p...

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Detalles Bibliográficos
Autores principales: Jin, Yue, Shen, Yan, Su, Xuan, Weintraub, Neal L., Tang, Yaoliang
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Research Network of Computational and Structural Biotechnology 2020
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7132053/
https://www.ncbi.nlm.nih.gov/pubmed/32280431
http://dx.doi.org/10.1016/j.csbj.2020.03.012