Figure 7.

Cartoon Highlighting the Applications of Myogenic Progenitors Described Here

Human iPSCs derived from healthy controls or patients are used as starting cells. Gene correction is applied to iPSCs using CRISPR/Cas9-mediated insertion of a cDNA into a safe harbor. Original or gene-corrected iPSCs are differentiated into the myogenic lineage using a 35-day transgene-free protocol. Myogenic progenitors are purified using a 1-step FACS procedure, and are then expanded (up to 5 × 10¹¹-fold) and cryopreserved. During expansion, purified myogenic progenitors are differentiated in vitro into myotubes with high fusion index, and show striation and spontaneous contraction upon in vitro maturation. Upon engraftment in immunodeficient mice, purified and expanded myogenic progenitors form human mononuclear cells and contribute to myofiber formation in vivo.