Shan Xiao-Qian, He Mei-Heng, Gao Wei-Li, Li Yu-Jiao, Liu Shu-Zhen, Liu Yan, Wang Chao-Li, Zhao Lan, Xu Shi-Xin
First Teaching Hospital of Tianjin University of Traditional Chinese Medicine, National Clinical Research Center for Chinese Medicine Acupuncture and Moxibustion, Tianjin 300381, China.
World J Stem Cells. 2025 Aug 26;17(8):109006. doi: 10.4252/wjsc.v17.i8.109006.
Alzheimer's disease (AD) is a multifaceted neurodegenerative disease for which effective disease-modifying therapies are lacking. Mesenchymal stem cell-derived exosomes (MSC-Exos) have emerged as a promising therapeutic approach due to their unique biological functions and favorable biocompatibility. This review systematically explores the mechanism of action of MSC-Exos in AD therapy, including the removal of β-amyloid the delivery of degradative enzymes, modulation of neuroinflammation, and promotion of neural regeneration. Meanwhile, this paper summarizes recent advances in preclinical and clinical studies, and analyzes the challenges in production standardization, safety assessment, and long-term efficacy validation of exosome therapies. Finally, several innovative strategies are proposed to enhance the therapeutic potential of MSC-Exos, including exosome functionalization and targeting optimization, gene editing techniques. This aims to promote the translation of exosomes from basic research to clinical application.
阿尔茨海默病（AD）是一种多方面的神经退行性疾病，目前缺乏有效的疾病修饰疗法。间充质干细胞衍生的外泌体（MSC-Exos）因其独特的生物学功能和良好的生物相容性，已成为一种有前景的治疗方法。本综述系统地探讨了MSC-Exos在AD治疗中的作用机制，包括清除β-淀粉样蛋白、递送降解酶、调节神经炎症和促进神经再生。同时，本文总结了临床前和临床研究的最新进展，并分析了外泌体疗法在生产标准化、安全性评估和长期疗效验证方面的挑战。最后，提出了几种创新策略来增强MSC-Exos的治疗潜力，包括外泌体功能化和靶向优化、基因编辑技术。这旨在促进外泌体从基础研究向临床应用的转化。
