Amongst the large variety of heritable muscular dystrophies, Duchenne muscular dystrophy (DMD) is the most common, affecting one in 3600 boys due to a mutation in the dystrophin gene.3, 4 Mouse models bearing mutations similar to those described in human muscular dystrophies, such as the dystrophin mutation in DMD, have been employed to develop myoblast transfer therapy (MTT) against muscular dystrophies. The gene discussed is DMD; the disease is muscular dystrophy.