Skeletal muscle progenitor cell therapy using iPSC-derived CD54-positive myogenic progenitors

The provided “target” is not a single molecule or receptor but a cell therapy approach: human induced pluripotent stem cell (iPSC)-derived myogenic progenitor cells enriched by the surface marker CD54 (ICAM-1), sometimes in combination with integrin α9β1 and syndecan-2, for skeletal muscle regeneration via engraftment and differentiation. CD54 was identified as a PAX7 target and enables prospective isolation of PAX7-induced myogenic progenitors from human pluripotent stem cell cultures in a GMP-compatible manner; CD54+ (±α9β1, SDC2) cells engraft injured mouse muscle, generate human dystrophin- and human lamin A/C–positive fibers, and can persist long term, with evidence of repopulating the satellite cell niche. Pluripotent stem cell–derived myogenic progenitors remodel their prenatal-like transcriptional state after in vivo engraftment, converging toward adult satellite cell signatures and showing regenerative potential comparable to adult satellite cells in mouse models. Broader reviews note historical limitations of myoblast/MuSC transplants (poor survival/migration) and the need for optimized differentiation, selection markers (e.g., CD54, ERBB3, NGFR), and delivery strategies for effective skeletal muscle repair, including applications to Duchenne muscular dystrophy and injury.

Other names

iPSC-derived CD54+ myogenic progenitor cellsCD54+ (ICAM-1+) myogenic progenitors derived from pluripotent stem cellsPAX7-induced iPSC myogenic progenitors enriched by CD54iPSC-derived skeletal muscle progenitor cells (SMPCs)ERBB3+ NGFR+ PAX7+ hPSC SMPCs

Cell replacement therapy through engraftment, fusion, and differentiation into skeletal myofibers Repopulation of the satellite cell compartment to sustain regeneration