Varshney Vibhav, Gabble Baneen C, Bishoyi Ashok Kumar, Varma Pooja, Qahtan Sarraa Ahmad, Kashyap Aditya, Panigrahi Rajashree, Nathiya Deepak, Chauhan Ashish Singh
Division of Pharmacology, Institute of Pharmaceutical Research, GLA University, Mathura, India.
Medical Laboratory Technique College, the Islamic University, Najaf, Iraq.
Mol Neurobiol. 2025 May 10. doi: 10.1007/s12035-025-05026-w.
Neurodegenerative diseases (NDs), like Alzheimer's disease (AD), Parkinson's disease (PD), Huntington's disease (HD), and Amyotrophic Lateral Sclerosis (ALS), present an increasingly significant global health burden, primarily due to the lack of effective early diagnostic tools and treatments. Exosomes-nano-sized extracellular vesicles secreted by nearly all cell types-have emerged as promising candidates for both biomarkers and therapeutic agents in NDs. This review examines the biogenesis, molecular composition, and diverse functions of exosomes in NDs. Exosomes play a crucial role in mediating intercellular communication. They are capable of reflecting the biochemical state of their parent cells and have the ability to cross the blood-brain barrier (BBB). In doing so, they facilitate the propagation of pathological proteins, such as amyloid-beta (Aβ), tau, and alpha-synuclein (α-syn), while also enabling the targeted delivery of neuroprotective compounds. Recent advancements in exosome isolation and engineering have opened up new possibilities for diagnostic and therapeutic strategies. These range from the discovery of non-invasive biomarkers to innovative approaches in gene therapy and drug delivery systems. However, challenges related to standardization, safety, and long-term effects must be addressed before exosomes can be translated into clinical applications. This review highlights both the promising potential and the obstacles that must be overcome to leverage exosomes in the treatment of NDs and the transformation of personalized medicine.
神经退行性疾病（NDs），如阿尔茨海默病（AD）、帕金森病（PD）、亨廷顿病（HD）和肌萎缩侧索硬化症（ALS），给全球健康带来了日益严重的负担，这主要是由于缺乏有效的早期诊断工具和治疗方法。外泌体——几乎所有细胞类型分泌的纳米级细胞外囊泡——已成为神经退行性疾病中有前景的生物标志物和治疗剂候选物。本综述探讨了外泌体在神经退行性疾病中的生物发生、分子组成和多种功能。外泌体在介导细胞间通讯中起关键作用。它们能够反映其母细胞的生化状态，并具有穿越血脑屏障（BBB）的能力。在此过程中，它们促进了病理性蛋白质的传播，如β-淀粉样蛋白（Aβ）、tau蛋白和α-突触核蛋白（α-syn），同时也实现了神经保护化合物的靶向递送。外泌体分离和工程方面的最新进展为诊断和治疗策略开辟了新的可能性。这些包括从发现非侵入性生物标志物到基因治疗和药物递送系统的创新方法。然而，在将外泌体转化为临床应用之前，必须解决与标准化、安全性和长期影响相关的挑战。本综述强调了利用外泌体治疗神经退行性疾病和实现个性化医疗的潜在前景以及必须克服的障碍。
