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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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Bibliografski detalji
Izdano u:Comput Struct Biotechnol J
Glavni autori: Jin, Yue, Shen, Yan, Su, Xuan, Weintraub, Neal L., Tang, Yaoliang
Format: Artigo
Jezik:Inglês
Izdano: Research Network of Computational and Structural Biotechnology 2020
Teme:
Online pristup:https://ncbi.nlm.nih.gov/pmc/articles/PMC7132053/
https://ncbi.nlm.nih.gov/pubmed/32280431
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.csbj.2020.03.012
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