Among the many diseases caused by gene mutations, Duchenne muscular dystrophy (DMD) is an intractable disease associated with severe outcomes. The patient’s body is unable to synthesize a protein called dystrophin, which leads to the loss of muscle tissue. With few options available to cure the disease, hopes are currently pinned on a combination of genome editing to correct the causative gene mutation, therapy to restore normal protein synthesis, and transplantions of cells derived from induced pluripotent stem cells (iPS cells).