Myostatin在杜氏肌营养不良疾病中的研究进展

Progress of myostatin on duchenne muscular dystrophy

杜氏肌营养不良(Duchenne muscular dystrophy,DMD)是一种严重的遗传性肌肉疾病,是由编码抗肌萎缩蛋白(dystrophin)的基因突变引起的。临床主要表现为肌肉进行性萎缩。Myostatin作为骨骼肌生长发育的负性调控因子,近年来其在肌肉萎缩性疾病中受到了广泛关注。该文就Myostatin结构、活性及其在骨骼肌生长发育中作用并对其抑制剂在DMD疾病中研究进展进行综述。

Duchenne muscular dystrophy(DMD)is a severe muscular genetic disorder, which is caused by a mutation in the DMD gene encoding the dystrophin protein.The clinical manifestation of DMD is the progressive muscular dystrophy.Myostatin is a negative regulator of skeletal muscle growth and development.In recent years, it has received extensive attention in the treatment of muscle diseases.This review focuses on the structural and activity of myostatin, its regulation of skeletal muscle growth and noval approaches to Myostatin inhibitor in the treatment of DMD.