Direct therapeutic targeting of dystrophin deficiency is challenged by the need to target large muscle mass, the extreme size of the DMD gene, 90 times larger than average and extensive genetic alterations, with a mutation rate exceeding that of other human genes (1.10−4 vs. 10−5–10−6) [10]. Here, DMD is linked to neuromuscular disease caused by qualitative or quantitative defects of dystrophin.