Abstract
Duchenne muscular dystrophy (DMD) is a rare genetic, progressive and devastating skeletal and cardiac muscle disorder due to mutation of the dystrophin gene that affects 1 in 3500 young males. Currently, there is no curative management for this pathology. The development of inducedpluripotent stem cells (iPSCs) offers a promising cell-based strategy for the treatment of muscular dystrophy. Several techniques have been established to generate functional myogenic progenitor cells derived from iPSCs. In addition, technologies in genetic modification using ZFN, TALENs, or CRISPR/Cas9 demonstrate potent methods to restore dystrophin expression. However, current evidence shows that either iPSCs or gene editing carry a risk of oncogenesis caused by the integration of exogenous DNA into the recipient gene. Thus, the safety issue is a major challenge for translating this method into human clinical applications. This review briefly discussed recent developments and progressions of iPSCs as well as genome engineering technologies relevant to regenerative medicine, especially for the treatment of DMD.
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