Promising therapeutic approach for Duchenne muscular dystrophy-sarcolipin reduction
This result entitled by “Reducing sarcolipin expression mitigates Duchenne muscular dystrophy and associated cardiomyopathy in mice” was published online on Oct 20th in the journal of Nature Communications. In the study, the authors found that sarcolipin (SLN), an inhibitor of SERCA, is abnormally upregulated in the dystrophin mutant (mdx) and dystrophin /utrophin double mutant mouse models of DMD. However, whether SLN upregulation underlies SERCA dysfunction and conduces to the muscle pathogenesis in DMD still needs to be clarified. To identify the physiological relevance of SLN upregulation with SERCA dysfunction, the scientists knocked down SLN with AAV9-mediated RNA interference approach, showing that loss function of SLN by AAV9 restores SERCA function in DMD mouse models. Meanwhile, muscle pathology is obviously ameliorated, the diaphragm, skeletal muscle and cardiac function are greatly improved. As such, this study discovered an effective means, i.e. SLN reduction, to mitigate Duchenne muscular dystrophy, which not only improves the SERCA function but also ameliorates the features of muscular dystrophy and cardiomyopathy, providing a promising therapeutic approach for DMD.
1. Duchenne muscular dystrophy.
2. Review of Duchenne muscular dystrophy (DMD) for the pediatricians in the community.
3. Reducing sarcolipin expression mitigates Duchenne muscular dystrophy and associated cardiomyopathy in mice.