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用于神经退行性疾病的纳米技术:脑靶向递送、刺激响应平台和细胞器特异性治疗的最新进展

Nanotechnology for Neurodegenerative Diseases: Recent Progress in Brain-Targeted Delivery, Stimuli-Responsive Platforms, and Organelle-Specific Therapeutics.

作者信息

Gao Lei, Wang Jia, Bi Yanhua

机构信息

Medical Imaging Department, Huabei Petroleum Administration Bureau General Hospital, Renqiu, Hebei, People's Republic of China.

Neurosurgery Department, Huabei Petroleum Administration Bureau General Hospital, Renqiu, Hebei, People's Republic of China.

出版信息

Int J Nanomedicine. 2025 Sep 9;20:11015-11044. doi: 10.2147/IJN.S549893. eCollection 2025.

Abstract

Neurodegenerative diseases-including Alzheimer's disease, Parkinson's disease, Huntington's disease, and amyotrophic lateral sclerosis-are characterized by progressive neuronal loss and complex pathological mechanisms such as protein aggregation, mitochondrial dysfunction, and neuroinflammation. Conventional therapies offer limited efficacy due to the blood-brain barrier (BBB) and lack of targeted delivery. Nanotechnology has emerged as a transformative strategy for precise brain-targeted treatment. This review summarizes recent advances in nanoparticle-based drug delivery systems, including polymeric nanoparticles, liposomes, inorganic nanomaterials, and biomimetic carriers, highlighting their design features, BBB-penetration mechanisms, and disease-specific applications. Emphasis is placed on stimuli-responsive nanocarriers that react to pH, reactive oxygen species, or enzyme activity, enabling site-specific drug release. Additionally, organelle-targeting strategies-particularly those directed at mitochondria and lysosomes-are explored for their role in subcellular precision therapy. The integration of diagnostic and therapeutic modalities in theranostic nanoplatforms is also discussed. By consolidating preclinical progress and emerging technologies, this review offers insights into the future of nanomedicine in treating neurodegenerative diseases and lays the groundwork for clinical translation.

摘要

神经退行性疾病,包括阿尔茨海默病、帕金森病、亨廷顿舞蹈症和肌萎缩侧索硬化症,其特征是神经元进行性丧失以及诸如蛋白质聚集、线粒体功能障碍和神经炎症等复杂的病理机制。由于血脑屏障(BBB)和缺乏靶向递送,传统疗法的疗效有限。纳米技术已成为一种用于精确脑靶向治疗的变革性策略。本综述总结了基于纳米颗粒的药物递送系统的最新进展,包括聚合物纳米颗粒、脂质体、无机纳米材料和仿生载体,突出了它们的设计特点、血脑屏障穿透机制以及针对特定疾病的应用。重点介绍了对pH、活性氧或酶活性作出反应的刺激响应性纳米载体,其能够实现药物的位点特异性释放。此外,还探讨了细胞器靶向策略,特别是针对线粒体和溶酶体的靶向策略在亚细胞精准治疗中的作用。还讨论了诊断与治疗模式在治疗诊断纳米平台中的整合。通过整合临床前进展和新兴技术,本综述为纳米医学在治疗神经退行性疾病方面的未来发展提供了见解,并为临床转化奠定了基础。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/029d/12433253/3d599523f0b9/IJN-20-11015-g0001.jpg

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