干细胞治疗肌萎缩侧索硬化症:细胞来源、数量、修饰手段及给药途径  

Stem cell therapy for amyotrophic lateral sclerosis:cell source,number,modification,and administration route

作　　者：赵文[1] 毕玉琳 付旭阳 段红梅[1] 杨朝阳[1] 李晓光[1,2] Zhao Wen;Bi Yulin;Fu Xuyang;Duan Hongmei;Yang Zhaoyang;Li Xiaoguang(Department of Neurobiology,School of Basic Medical Sciences,Capital Medical University,Beijing 100069,China;Beijing Key Laboratory for Biomaterials and Neural Regeneration,School of Biological Science and Medical Engineering,Beihang University,Beijing 100083,China)

机构地区：[1]首都医科大学基础医学院神经生物学系,北京市100069 [2]北京航空航天大学生物与医学工程学院,生物材料与神经再生北京市重点实验室,北京市100083

出　　处：《中国组织工程研究》2025年第19期4083-4090,共8页Chinese Journal of Tissue Engineering Research

基　　金：国家自然科学基金(82271403),项目负责人:李晓光;国家自然科学基金(82272171),项目负责人:杨朝阳;北京市自然科学基金(7222004),项目负责人:段红梅。

摘　　要：背景:随着医疗技术的不断进步,干细胞疗法已经被用于包括肌萎缩侧索硬化症在内的多种疾病的治疗。目的:综述干细胞治疗肌萎缩侧索硬化症的研究进展,展望该领域的发展趋势。方法:以“肌萎缩侧索硬化症,间充质干细胞,神经干/祖细胞,多能诱导干细胞;amyotrophic lateral sclerosis,mesenchymal stem cells,neural stem/progenitor cells,pluripotent stem cells”为关键词,在PubMed、中国知网、万方数据库进行检索,检索时限为1995-2024年。共检索到1700余篇文献,最终纳入58篇文献用于综述。结果与结论:肌萎缩侧索硬化症是一种影响脑干和脊髓的下部运动神经元以及运动皮质的上部运动神经元的神经退行性疾病。干细胞治疗肌萎缩侧索硬化症的相关研究已成为一大研究热点。在这篇综述中,总结了不同种类干细胞在肌萎缩侧索硬化症研究领域中的应用情况,包括间充质干细胞、神经干祖细胞、多能诱导干细胞,评估了干细胞来源、细胞量、干细胞修饰手段及给药途径等临床前研究关键,为未来干细胞治疗的应用奠定了基础。BACKGROUND:With the continuous advancement of medical technology,stem cell therapy has been used to treat a variety of diseases,including amyotrophic lateral sclerosis.OBJECTIVE:To review the research progress of stem cell therapy for amyotrophic lateral sclerosis,and prospect the development trend of this field.METHODS:PubMed,China National Knowledge Infrastructure(CNKI),and WanFang Data were searched for articles published from 1995 to 2024 using the key words“amyotrophic lateral sclerosis,mesenchymal stem cells,neural stem/progenitor cells,pluripotent stem cells.”A total of more than 1700 articles were retrieved,and 58 articles were finally included in this review.RESULTS AND CONCLUSION:Amyotrophic lateral sclerosis is a neurodegenerative disease that affects lower motor neurons in the brainstem and spinal cord and upper motor neurons in the motor cortex.The related research of stem cells in the treatment of amyotrophic lateral sclerosis has become a research hotspot.In this review,we summarize the application of different types of stem cells in amyotrophic lateral sclerosis research,including mesenchymal stem cells,neural stem progenitor cells,and induced pluripotent stem cells,and evaluate the key points of preclinical research such as stem cell source,cell volume,stem cell modification methods,and drug delivery routes,which lays the foundation for the future application of stem cell therapy.

关 键 词：肌萎缩侧索硬化症 间充质干细胞 神经干/祖细胞 诱导多能干细胞 神经退行性疾病 

分 类 号：R459.9[医药卫生—治疗学] R318[医药卫生—临床医学] R744.8