Functional dystrophin gene restoration has been demonstrated by CRISPR/Cas9 editing in myoblasts differentiated from induced pluripotent stem cells (iPSCs) of DMD patients [99, 100] [described in more detail in “Generation of myoblasts from embryonic stem cells (ESCs) and induced pluripotent stem cells (iPSCs)”]. The gene discussed is DMD; the disease is Duchenne muscular dystrophy.