帕金森病靶向治疗研究进展  

Advances in targeted therapies for Parkinson’s disease

作　　者：蒋政 欧汝威 商慧芳[1] 宋伟[1] JIANG Zheng;OU Ru-wei;SHANG Hui-fang;SONG Wei(Department of Neurology,West China Hospital,Sichuan University,Chengdu 610041,Sichuan,China)

机构地区：[1]四川大学华西医院神经内科

出　　处：《中国现代神经疾病杂志》2019年第11期902-908,共7页Chinese Journal of Contemporary Neurology and Neurosurgery

基　　金：国家老年疾病临床医学研究中心（四川大学华西医院）基础研究重点资助项目（项目编号：Z2018B08)~~

摘　　要：帕金森病是临床常见神经变性病。虽然目前多巴胺替代治疗仍是帕金森病治疗的基石,但受到运动并发症和远期疗效的制约。20余年来,帕金森病遗传学研究的快速发展使得疾病修饰疗法成为可能,开发靶向药物阻断异常分子通路业已成为当前帕金森病研究的热点。本文针对SNCA、GBA和LRRK2基因突变,以及受其影响分子通路靶向治疗的最新进展进行综述,以期提高临床对帕金森病精准化治疗的认识。Parkinson’s disease(PD) is the second most common neurodegenerative disease.Although dopamine replacement therapy is the golden standard of PD treatment, there are limitations due to drug-related motor complications and loss of long-term efficacy. In the recent two decades, rapid advances in the genetics of PD have made it possible to develop disease-modifying therapies. The development of targeted drugs blocking involved pathogenic pathways has become a hot research area in PD. This review summarizes the recent advances in targeted therapies based on three genetic mutations of SNCA, GBA and LRRK2 for PD and involved pathways to improve clinicians’ understanding on the precision treatment in PD.

关 键 词：帕金森病 药物疗法 基因 突变 综述 

分 类 号：R74[医药卫生—神经病学与精神病学]