Perlingeiro lab uses gene editing to correct mutations in induced pluripotent stem cells (iPSCs) generated from patients with muscular dystrophy.

The Perlingeiro lab has used CRISPR-Cas9 technology to correct mutations in induced pluripotent stem cells (iPSCs) generated from patients with muscular dystrophy.  Myogenic progenitors derived from these gene corrected iPSCs were able to engraft into dystrophic mice, giving rise to donor-derived muscle fibers with rescued function as well as muscle stem cells.  These results suggest that gene editing of patient-specific iPSCs could be used for cell transplantation therapies in the future.  Read the paper.

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