Humanizing the mdx mouse model of DMD: the long and the short of it

Components of the dystrophin-associated glycoprotein complex. The dystrophin-associated glycoprotein complex (DGC) and proteins that interact with DGC are depicted here. Extracellular, membrane, cytoplasmic and nuclear components are shown. The proteins targeted in double knockout mouse models of DMD, as outlined in the text, are indicated: (1) Dystrophin (2) Utrophin (3) α-Dystrobrevin (4) α7-Integrin (5) Myogenic differentiation factor 1(Myod) (6) Glycans and (7) telomere length. The domains of dystrophin are also indicated: (i) N-terminal domain (ii) middle body domain, which includes the nNOS binding sites (iii) cysteine-rich domain and (iv) C-terminal domain. In addition, DMD disease modifiers not directly involved in the DGC but that are also targeted in double knockout studies are also highlighte