帕金森病细胞替代治疗的现状及进展

doi:10.20223/j.cnki.1000-8535.2025.12.001

广州医药 ›› 2025, Vol. 56 ›› Issue (12): 1611-1620.DOI: 10.20223/j.cnki.1000-8535.2025.12.001

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帕金森病细胞替代治疗的现状及进展

陈洁纯, 黎明涛, 马珊珊

中山大学中山医学院,广东省脑功能与脑疾病重点实验室（广东广州 510080）

收稿日期:2025-01-03 出版日期:2025-12-20 发布日期:2026-01-20
通讯作者: 马珊珊,E-mail： mashsh3@mail.sysu.edu.cn;黎明涛,E-mail： limt@mail.sysu.edu.cn
作者简介:马珊珊, 医学博士,中山大学中山医学院副教授,中山医学院药理教研室秘书、广东省脑功能与脑疾病重点实验室秘书。2013年获中山大学医学博士学位,曾获“中山大学博士生国外访学与国际合作项目”赴美国匹兹堡大学医学中心交流学习。研究方向为帕金森病发病机制及治疗新策略,以第一或通讯作者发表多篇SCI论文,包括eLife,Neurobiol Dis,J Biol Chem,Cell Death Dis等国际主流期刊。以负责人获国家自然科学基金青年项目、面上项目,广东省自然科学基金面上项目,广州市科技计划项目等资助。
黎明涛, 中山大学中山医学院二级教授、博士生导师,广东省脑功能与脑疾病重点实验室主任,蛋白质组学转化医学广东省普通高校重点实验室主任,享受国务院特殊津贴专家荣誉。专注于帕金森病多巴胺能神经元死亡机制及防治新靶标研究。作为第一完成人获得2011年“教育部自然科学奖”一等奖和2011年“广东省科学技术奖”一等奖。发表SCI论文40余篇,其中包括J Neurosci,eLife,Mol Cell Biol,J Biol Chem等国际主流期刊。作为主持人共获得38项基金,包括国家自然科学基金重点项目、国家自然科学基金B类杰青、国家自然科学基金-广东联合资助基金（集成项目1项,重点项目2项）、国家自然科学基金面上项目、“重大新药创制”科技重大专项、“973”计划、广东省科技厅重点领域研发计划“脑科学与类脑研究专项”、广东省自然团队项目等。
基金资助:
广东省重点领域研发计划“脑科学与类脑研究”专项（2018B030337001）; 广东省脑功能与脑疾病重点实验室（2023B1212060018）

Current status and advances in cell replacement therapy for Parkinson's disease

CHEN Jiechun, LI Mingtao, MA Shanshan

Guangdong Provincial Key Laboratory of Brain Function and Disease,Zhongshan School of Medicine,Sun Yat-sen University,Guangzhou 510080,China

Received:2025-01-03 Online:2025-12-20 Published:2026-01-20

摘要/Abstract

摘要： 帕金森病是全球第二大神经退行性疾病,其根本病理特征为中脑黑质多巴胺能神经元的退变死亡。目前临床一线治疗主要采用左旋多巴替代疗法,然而疗效有限且副作用显著。以补充多巴胺能神经元为基础的细胞替代疗法,能够从根本上解决神经元丢失的问题,具有长远的临床意义。细胞替代治疗的细胞最早来自于胎儿腹侧中脑组织,随着重编程技术的不断发展,已逐步转向人类胚胎干细胞和诱导多能干细胞体外分化的前体细胞。同时,直接在体内重编程,将胶质细胞转分化为多巴胺能神经元,也是一种具有应用潜力的策略。本文系统总结了近年来帕金森病细胞替代疗法的进展和面临的挑战,旨在为该疾病治疗新策略的研究提供参考和启示。

关键词: 帕金森病, 多巴胺, 细胞替代疗法, 重编程

Abstract: Parkinson's disease（PD）is the second most prevalent neurodegenerative disorder worldwide,characterized by the progressive loss of dopaminergic neurons in the substantia nigra pars compacta．Traditional treatments,primarily involving levodopa for dopamine replacement,offer limited efficacy and associated with significant side effects．Cell replacement therapies aimed at replenishing dopaminergic neurons provide a promising long-term solution to neuronal loss,with substantial clinical significance．The initial successful cellular source for transplantation in PD research was fetal ventral mesencephalic tissue．Nevertheless,advancements in reprogramming technologies have increasingly favored the use of human embryonic stem cells and induced pluripotent stem cells．Additionally,direct in vivo reprogramming,converting glial cells into dopaminergic neurons,has emerged as an alternative strategy for cell replacement therapy．This review systematically summarizes the recent advances and challenges in cell replacement therapies for PD,with the aim of providing insights and guidance for the development of novel therapeutic strategies for the PD．

Key words: Parkinson's disease, dopamine, cell replacement therapy, reprogram

陈洁纯, 黎明涛, 马珊珊. 帕金森病细胞替代治疗的现状及进展[J]. 广州医药, 2025, 56(12): 1611-1620.

CHEN Jiechun, LI Mingtao, MA Shanshan. Current status and advances in cell replacement therapy for Parkinson's disease[J]. Guangzhou Medical Journal, 2025, 56(12): 1611-1620.

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帕金森病细胞替代治疗的现状及进展

Current status and advances in cell replacement therapy for Parkinson's disease

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