Hu Ningyun, Chen Liang, Hu Guoku, Ma Rong
Department of Pharmacology and Experimental Neuroscience, University of Nebraska Medical Center, Omaha, NE 68198-5880, USA.
Millard West High School, Omaha, NE 68135, USA.
Explor Neuroprotective Ther. 2025;5. doi: 10.37349/ent.2025.1004104. Epub 2025 May 6.
Neurodegenerative diseases represent a significant and growing challenge to public health worldwide. Current therapeutic strategies often fall short in halting or reversing disease progression, highlighting the urgent need for novel approaches. Extracellular vesicles (EVs) have garnered attention as potential therapeutic agents due to their role in intercellular communication and their ability to transport bioactive cargo, including proteins, nucleic acids, and lipids. This review provides a comprehensive overview of the biology of EVs, their involvement in neurodegenerative diseases, and the potential for EV-based therapies. We discuss the different types of EVs, their biogenesis, and their cargo composition, emphasizing their relevance to neurological processes such as protein misfolding, neuroinflammation, and oxidative stress. Preclinical studies investigating EVs as carriers of therapeutic cargo and their ability to promote neuronal survival and regeneration are examined, with a focus on evidence from animal models of neurodegenerative disorders. We explore the use of EVs in the treatment of neurodegenerative diseases, including ongoing clinical trials, methods for EV isolation and modification, and future perspectives on personalized EV-based therapies designed to meet the unique needs of individual patients. Overall, this review highlights the potential of EVs as a promising avenue for neurodegenerative disease therapy, while also addressing key research gaps and translational hurdles that need to be overcome for their successful clinical implementation.
神经退行性疾病对全球公共卫生构成了重大且日益严峻的挑战。当前的治疗策略往往在阻止或逆转疾病进展方面效果不佳,凸显了对新方法的迫切需求。细胞外囊泡(EVs)因其在细胞间通讯中的作用以及运输生物活性物质(包括蛋白质、核酸和脂质)的能力而作为潜在治疗剂受到关注。本综述全面概述了EVs的生物学特性、它们在神经退行性疾病中的作用以及基于EVs的治疗潜力。我们讨论了不同类型的EVs、它们的生物发生过程以及它们的货物组成,强调了它们与蛋白质错误折叠、神经炎症和氧化应激等神经学过程的相关性。研究了将EVs作为治疗货物载体及其促进神经元存活和再生能力的临床前研究,重点关注神经退行性疾病动物模型的证据。我们探讨了EVs在神经退行性疾病治疗中的应用,包括正在进行的临床试验、EVs分离和修饰方法以及旨在满足个体患者独特需求的个性化基于EVs的治疗的未来前景。总体而言,本综述强调了EVs作为神经退行性疾病治疗有前景途径的潜力,同时也解决了其成功临床应用需要克服的关键研究差距和转化障碍。