EXPLORING THE ROLE OF PHOSPHODIESTERASE PDE5 INHIBITION IN THE TREATMENT OF MUSCULAR DYSTROPHY

摘要

Duchenne muscular dystrophy (DMD) is caused by a mutation of a single gene, dystrophin, and is characterized by a slow progression and the increased turnover of myofiber degeneration and regeneration. Despite the long history of scientific research, the full mechanisms of muscle damage still need to be clarified and the therapeutic choices are limited. Recent studies on blood flow regulation have led to the discovery of a novel pharmacological treatment for muscular dystrophies. Phosphodiesterase PDE5 inhibitors were originally designed to improve blood circulation by inducing vasodilation in target organs and are widely used to treat male erectile dysfunction. Recently, the range of potential clinical applications for PDE5 inhibitors has widened, with increasing reports of their efficacy against various conditions, including pulmonary hypertension. The potential efficacy against DMD was reported in animal studies using the murine equivalent model. In this review, I discuss the recent understanding of the role of blood flow regulation in the pathogene-sis of muscular dystrophy. Therapeutic approaches with PDE5 inhibitors will be discussed and include citations from the recent publications with animal studies. Future considerations of PDE5 inhibitor therapies will be examined, including the evaluation of drug efficacy and potential side effects.