DMD and Duchenne muscular dystrophy: 2012). The internally truncated dystrophin isoforms encoded by the manipulated transcripts contain the necessary N- and C-terminal domains that allow interaction with cytoskeletal actin and the dystrophin-associated protein complex (for review see Cohn and Campbell 2000). The production of even low levels of these BMD-like dystrophin isoforms is postulated to allow partial restoration of the dystrophin-associated protein complex, decreasing the rate of muscle fiber degeneration and conferring a degree of clinical benefit in DMD patients (Mendell et al. 2013).