Liu Shuhan, Li Haisong, Xi Shiwen, Zhang Yuning, Sun Tianmeng
Key Laboratory of Organ Regeneration and Transplantation of Ministry of Education, Institute of Immunology, The First Hospital, Jilin University, Changchun, Jilin, People's Republic of China.
National-Local Joint Engineering Laboratory of Animal Models for Human Diseases, Changchun, People's Republic of China.
Int J Nanomedicine. 2025 Feb 4;20:1443-1490. doi: 10.2147/IJN.S457393. eCollection 2025.
Given the complexity of the central nervous system (CNS) and the diversity of neurological conditions, the increasing prevalence of neurological disorders poses a significant challenge to modern medicine. These disorders, ranging from neurodegenerative diseases to psychiatric conditions, not only impact individuals but also place a substantial burden on healthcare systems and society. A major obstacle in treating these conditions is the blood-brain barrier (BBB), which restricts the passage of therapeutic agents to the brain. Nanotechnology, particularly the use of nanoparticles (NPs), offers a promising solution to this challenge. NPs possess unique properties such as small size, large surface area, and modifiable surface characteristics, enabling them to cross the BBB and deliver drugs directly to the affected brain regions. This review focuses on the application of NPs in gene therapy and enzyme replacement therapy (ERT) for neurological disorders. Gene therapy involves altering or manipulating gene expression and can be enhanced by NPs designed to carry various genetic materials. Similarly, NPs can improve the efficacy of ERT for lysosomal storage disorders (LSDs) by facilitating enzyme delivery to the brain, overcoming issues like immunogenicity and instability. Taken together, this review explores the potential of NPs in revolutionizing treatment options for neurological disorders, highlighting their advantages and the future directions in this rapidly evolving field.
鉴于中枢神经系统（CNS）的复杂性和神经疾病的多样性，神经疾病患病率的不断上升对现代医学构成了重大挑战。这些疾病，从神经退行性疾病到精神疾病，不仅影响个人，也给医疗系统和社会带来了沉重负担。治疗这些疾病的一个主要障碍是血脑屏障（BBB），它限制了治疗药物进入大脑。纳米技术，特别是纳米颗粒（NPs）的使用，为这一挑战提供了一个有前景的解决方案。纳米颗粒具有诸如尺寸小、表面积大以及可修饰的表面特性等独特性质，使其能够穿过血脑屏障并将药物直接递送至受影响的脑区。本综述聚焦于纳米颗粒在神经疾病的基因治疗和酶替代疗法（ERT）中的应用。基因治疗涉及改变或操纵基因表达，并且可以通过设计用于携带各种遗传物质的纳米颗粒得到增强。同样，纳米颗粒可以通过促进酶向大脑的递送，克服诸如免疫原性和不稳定性等问题，提高溶酶体贮积症（LSDs）的酶替代疗法的疗效。综上所述，本综述探讨了纳米颗粒在彻底改变神经疾病治疗选择方面的潜力，突出了它们的优势以及这个快速发展领域的未来方向。
