帕金森病发病机制与治疗研究进展

The mechanism underlying pathogenesis of Parkinson’s disease and the progress in its therapeutics

帕金森病是一种以多巴胺能神经元进行性死亡为主要病理特征的神经系统退行性病变,其临床表现为运动及非运动症状两种形式。我国帕金森病患者数量大概为500万,占世界上总帕金森病患者人数的一半。帕金森病的发病机制目前还不是很明确,主要观点认为与蛋白质异常聚集、线粒体功能障碍、氧化应激等相关。临床上帕金森病常用的治疗手段包括以左旋多巴为首的药物疗法、手术疗法和物理疗法。随着对帕金森病研究的深入以及生物医学领域研究进展,细胞替换法、基因疗法、脑深部电刺激越来越受到人们的重视。文章从帕金森病的发病机制出发,简要介绍其潜在的治疗靶点并归纳现有的治疗手段,为更好的认识及治疗帕金森病提供参考。

Parkinson′s disease(PD)is a neurodegenerative disorder,characterized by progressive loss of dopaminergic neurons in the substantia nigra. The clinical manifestations of PD include motor and non-motor symptoms. There are around 5 millions PD patients in China,accounting for half of the total PD cases in the world. The mechanisms underlying PD pathogenesis remain to be elucidated,but it is widely accepted that abnormal protein aggregation,mitochondria dysfunction,and oxidative stress are the key causative factors of PD. The regular treatment for PD includes medication of which L-dopa is the most commonly prescribed,surgical treatment,and physiotherapy. In recent year,with the progress in biotechnology and understanding of mechanisms underlying PD,novel therapeutics such as cell replacement,gene therapy,deep brain stimulation emerge,which substantially benefit the treatment of PD. In present review,based on the molecular mechanisms underlying PD pathogenesis,we summarized and discussed current therapeutic strategy on PD.