Many therapies aiming to restore dystrophin expression such as exon skipping or gene delivery are currently in the preclinical or early clinical phase.28 Despite their great therapeutic potential, gene therapies have led so far to mild dystrophin re-expression in DMD muscles and poor improvement in muscle phenotype owing to multiple technical issues such as lack of specificity, poor delivery and low efficiency. Here, DMD is linked to Duchenne muscular dystrophy.