MicroRNAs in Dystrophinopathy.
Ahyoung LeeJiwon MoonJin YuChangwon KhoPublished in: International journal of molecular sciences (2022)
Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD), which represent the range of dystrophinopathies, account for nearly 80% of muscle dystrophy. DMD and BMD result from the loss of a functional dystrophin protein, and the leading cause of death in these patients is cardiac remodeling and heart failure. The pathogenesis and progression of the more severe form of DMD have been extensively studied and are controlled by many determinants, including microRNAs (miRNAs). The regulatory role of miRNAs in muscle function and the differential miRNA expression in muscular dystrophy indicate the clinical significance of miRNAs. This review discusses the relevant microRNAs as potential biomarkers and therapeutic targets for DMD and DMD cardiomyopathy as examples of dystrophinopathies.
Keyphrases
- muscular dystrophy
- duchenne muscular dystrophy
- heart failure
- end stage renal disease
- skeletal muscle
- ejection fraction
- newly diagnosed
- poor prognosis
- early onset
- left ventricular
- chronic kidney disease
- binding protein
- peritoneal dialysis
- multidrug resistant
- transcription factor
- small molecule
- patient reported
- acute heart failure