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Genomic Safe Harbor Expression of PAX7 for the Generation of Engraftable Myogenic Progenitors

3 Dec 2020

Abstract excerpt

Inducible expression of PAX7 in differentiating pluripotent stem cells (PSCs) allows massively scalable generation of human myogenic progenitors, which upon transplantation into dystrophic muscles give rise to donor-derived myofibers and satellite cells. Therefore, PSC-derived PAX7 + myogenic progenitors represent an attractive therapeutic approach to promote muscle regeneration. Work to date has used lentiviral...

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PMID: 33275879
DOI: 10.1016/j.stemcr.2020.11.001

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