In the past decade, the development of two innovative technologies, namely, induced pluripotent stem cells (iPSCs) and the\nCRISPR Cas9 system, has enabled researchers to model diseases derived from patient cells and precisely edit DNA sequences of\ninterest, respectively. In particular, Duchenne muscular dystrophy (DMD) has been an exemplary monogenic disease model for\ncombining these technologies to demonstrate that genome editing can correct genetic mutations in DMD patient-derived iPSCs.\nDMD is an X-linked genetic disorder caused by mutations that disrupt the open reading frame of the dystrophin gene, which\nplays a critical role in stabilizing muscle cells during contraction and relaxation. The CRISPR Cas9 system has been shown to be\ncapable of targeting the dystrophin gene and rescuing its expression in in vitro patient-derived iPSCs and in vivo DMD mouse\nmodels. In this review, we highlight recent advances made using the CRISPR Cas9 system to correct genetic mutations and\ndiscuss how emerging CRISPR technologies and iPSCs in a combined platform can play a role in bringing a therapy for DMD\ncloser to the clinic.
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