DMD and Duchenne muscular dystrophy: Cell-based therapies were proposed for DMD by transplantation of myoblasts in which the enthusiasm of restoring dystrophin in the mdx mouse model by intra-muscular (IM) myoblast transplantation, resulted in 4 clinical trials in humans, that ultimately all failed to restore sufficient dystrophin to provide functional benefit (Partridge et al., 1989; Law et al., 1991; Gussoni et al., 1992; 1997; Huard et al., 1992; Karpati et al., 1993; Miller et al., 1997).