Researchers identify new targets for DMD

Two studies published in Nature Communications show that decreasing expression of nuclear factor I X (NFIX) or sarcolipin (SLN) improves phenotypes associated with Duchenne muscular dystrophy in mouse models of the disease, identifying new targets for the indication.

In one study, researchers from Rutgers University and colleagues found that SLN expression was elevated in cardiac and skeletal muscles of animal models of DMD, as well as in quadriceps and cardiac ventricles of human DMD patients. Both haploinsufficiency and genetic deletion of SLN in a DMD mouse model extended median lifespan by 446 and 368 days, respectively, compared with normal SLN expression. SLN knockout in DMD mice also reduced necrosis and fibrosis in the quadriceps and diaphragm and increased forelimb grip strength...