帕金森病细胞移植治疗和基因治疗进展

doi:10.3969/j.issn.1672-6731.2022.04.005

摘要/Abstract

摘要： 细胞移植治疗和基因治疗在帕金森病治疗中的作用越来越受到关注,已从实验室的研究工具发展为面向患者的临床级产品。细胞移植治疗的目的是替代退化的中脑多巴胺能神经元并恢复失神经支配的黑质纹状体通路多巴胺传递。基因治疗可通过引入遗传物质以延缓多巴胺能神经元退化,恢复多巴胺能信号转导;基于神经营养因子的基因治疗及其递送方式已进入临床试验阶段;基因治疗还可干预α-突触核蛋白介导的神经退行性变的病理过程。本文综述帕金森病细胞移植治疗和基因治疗最新进展,展望未来治疗策略。

关键词: 帕金森病, 细胞移植, 基因治疗, 神经生长因子, α 突触核蛋白, 综述

Abstract: The role of cell transplantation therapy and gene therapy in the treatment of Parkinson's disease (PD) has attracted more and more attention. It has developed from laboratory research tools to clinical products for patients. The purpose of cell transplantation therapy in PD is to replace degenerated midbrain dopamine neurons and restore dopamine neurotransmission in the denervation of the nigrostriatal pathway. Gene therapy works by introducing genetic material to slow down the degeneration of dopamine neurons and restore dopaminergic signaling transduction. Using neurotrophic factor and delivery methods for neurotrophic factor have entered clinical trials. It can also intervene in the pathological process of α - synuclein (α-Syn) mediated neurodegeneration. This paper reviews the latest progress of cell transplantation therapy and gene therapy for PD, and looks forward to the future treatment strategies.

Key words: Parkinson disease, Cell transplantation, Genetic therapy, Nerve growth factor, Alpha-synuclein, Review

许保磊, 陈彪. 帕金森病细胞移植治疗和基因治疗进展[J]. 中国现代神经疾病杂志, 2022, 22(4): 243-252.

XU Bao-lei, CHAN Piu. Advances in cell transplantation therapy and gene therapy in Parkinson's disease[J]. Chinese Journal of Contemporary Neurology and Neurosurgery, 2022, 22(4): 243-252.

http://journal11.magtechjournal.com/cjcnn/CN/Y2022/V22/I4/243

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