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

ABSTRACT: 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 vectors (LVs) that randomly integrate inducible PAX7 transgenes. Here, we investigated whether equivalent induction of the myogenic program could be achieved by targeting the PAX7 transgene into genomic safe harbor (GSH) sites. Across multiple PSC lines, we find that this approach consistently generates expandable myogenic progenitors in vitro, although scalability of exp

SUBMITTER: Kim H 

PROVIDER: S-EPMC7815936 | biostudies-literature | 2021 Jan

REPOSITORIES: biostudies-literature