These therapeutic strategies for DMD/BMD fall into four categories [9-12]: (1) mutation-specific therapeutic approaches for repairing the genetic defects or the transcripts (exon skipping, nonsense codon suppression and endonuclease-mediated gene correction); (2) gene replacement therapy, which can be applied to the entire patient population; (3) cell therapy including allogeneic stem cells with mutations corrected ex vivo; and (4) modulation of non-dystrophin gene expression such as upregulation of utrophin to compensate for the lack of dystrophin. This evidence concerns the gene DMD and Duchenne muscular dystrophy.