基于SNCA基因治疗帕金森病的研究进展  

Advances in Treatment of Parkinson’s Disease Based on SNCA Gene

作　　者：李昕 刘亦晨[1] 王攀[1] LI Xin;LIU Yichen;WANG Pan(College of Life Science,Shaanxi Normal University,Xi’an 710119,China)

机构地区：[1]陕西师范大学生命科学学院,西安710119

出　　处：《中国细胞生物学学报》2020年第10期1843-1851,共9页Chinese Journal of Cell Biology

基　　金：国家自然科学基金(批准号:81872497);中央高校基本科研业务费专项资金(批准号:2020CSLY013)资助的课题。

摘　　要：帕金森病是常见的威胁人类健康的神经退行性疾病之一,其主要病理特征为中脑黑质路易小体的形成和多巴胺能神经元的丢失,而路易小体的主要成分是α-突触核蛋白(α-synuclein,α-syn)。现已证实,编码α-syn的SNCA基因是帕金森病的关键致病基因之一,该基因的突变可引起α-syn的过量表达,进而形成路易小体,导致帕金森病的发生。因此,SNCA基因被认为是对帕金森病进行治疗的有效靶点。该文从SNCA基因在帕金森病发病中的作用与机制出发,讨论了SNCA基因与α-syn在帕金森病的发生和进行性恶化中的具体作用通路及过程,并从减少α-syn的表达水平、抑制α-syn聚集体的形成、促进α-syn聚集体的降解及抑制和阻断α-syn聚集体的传播等方面,总结了目前基于SNCA基因和α-syn调控的治疗方法,为开发帕金森病诊疗新策略提供参考。Parkinson’s disease is one of the common neurodegenerative diseases that threaten human health.Its main pathological features are the formation of midbrain nigra Lewy bodies and the loss of dopaminergic neurons,and the main component of Lewy bodies is α-synuclein.It has been confirmed that the SNCA gene encoding α-synuclein is one of the key pathogenic genes of Parkinson’s disease.The mutation of SNCA can cause the overexpression of α-synuclein,which in turn leads to the formation of Lewy bodies,leading to Parkinson’s disease.Therefore,SNCA gene can be considered as an effective target for the precise treatment of Parkinson’s disease.Based on the role and mechanism of SNCA gene in the pathogenesis of Parkinson’s disease,this paper discusses the specific pathway and process of SNCA gene and α-syn in the occurrence and progressive deterioration of Parkinson’s disease.The current treatment methods based on SNCA gene and α-syn regulation are summarized from the aspects of reducing the expression level of α-syn,inhibiting the formation of α-syn aggregates,promoting the degradation of α-syn aggregates,inhibiting and blocking the spread of α-syn aggregates,so as to provide reference for the development of new diagnostic and therapeutic strategies for Parkinson’s disease.

关 键 词：帕金森病 SNCA基因 Α-突触核蛋白 

分 类 号：R742.5[医药卫生—神经病学与精神病学]