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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...
| Autores principales: | , , , , |
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| Formato: | Online Artículo Texto |
| Lenguaje: | English |
| Publicado: |
Research Network of Computational and Structural Biotechnology
2020
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| 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 |