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用于递送小干扰RNA治疗神经退行性疾病的纳米技术:聚焦阿尔茨海默病和帕金森病

Nanotechnology used for siRNA delivery for the treatment of neurodegenerative diseases: Focusing on Alzheimer's disease and Parkinson's disease.

作者信息

Li Yan, Zhang Jiahui, Ma Boqin, Yu Wenjun, Xu Meixia, Luan Weijing, Yu Qinglong, Zhang Li, Rong Rong, Fu Yuanlei, Cao Haiqiang

机构信息

Yantai Key Laboratory of Nanomedicine & Advanced Preparations, Yantai Institute of Materia Medica, Shandong 264000, China; Shandong Laboratory of Yantai Drug Discovery, Bohai Rim Advanced Research Institute for Drug Discovery, Yantai, Shandong 264117, China.

School of Pharmacy, Yantai University, Yantai 264005, China.

出版信息

Int J Pharm. 2024 Dec 5;666:124786. doi: 10.1016/j.ijpharm.2024.124786. Epub 2024 Oct 6.

DOI:10.1016/j.ijpharm.2024.124786
PMID:39378955
Abstract

Neurodegenerative diseases (ND) are often accompanied by dementia, motor dysfunction, or disability. Caring for these patients imposes a significant psychological and financial burden on families. Until now, there are no effective methods for the treatment of NDs. Among them, Alzheimer's disease (AD) and Parkinson's disease (PD) are the two most common. Recently, studies have revealed that the overexpression of certain genes may be linked to the occurrence of AD and PD. Small interfering RNAs (siRNAs) are a powerful tool for gene silencing because they can specifically bind to and cleave target mRNA. However, the intrinsic properties of naked siRNA and various physiological barriers limit the application of siRNA in the brain. Nanotechnology is a promising option for addressing these issues. Nanoparticles are not only able to protect siRNA from degradation but also have the advantage of crossing various physiological barriers to reach the brain target of siRNA. In this review, we aim to introduce diverse nanotechnology used for delivering siRNA to treat AD and PD. Finally, we will briefly discuss our perspectives on this promising field.

摘要

神经退行性疾病(ND)常伴有痴呆、运动功能障碍或残疾。照顾这些患者给家庭带来了巨大的心理和经济负担。到目前为止,尚无治疗神经退行性疾病的有效方法。其中,阿尔茨海默病(AD)和帕金森病(PD)是最常见的两种。最近,研究表明某些基因的过度表达可能与AD和PD的发生有关。小干扰RNA(siRNA)是一种强大的基因沉默工具,因为它们可以特异性结合并切割靶mRNA。然而,裸siRNA的固有特性和各种生理屏障限制了siRNA在脑内的应用。纳米技术是解决这些问题的一个有前景的选择。纳米颗粒不仅能够保护siRNA不被降解,而且具有跨越各种生理屏障到达siRNA脑靶点的优势。在本综述中,我们旨在介绍用于递送siRNA治疗AD和PD的各种纳米技术。最后,我们将简要讨论我们对这个有前景领域的看法。

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