Induced pluripotent stem cells and genome editing technology as therapeutic strategies for Duchenne muscular dystrophy

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.