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基于小干扰RNA的纳米载体在神经退行性疾病治疗中的作用。

Role of siRNA-based nanocarriers for the treatment of neurodegenerative diseases.

作者信息

Mishra Neeraj, Ashique Sumel, Garg Ashish, Rai Vineet Kumar, Dua Kamal, Goyal Amit, Bhatt Shvetank

机构信息

Amity Institute of Pharmacy, Amity University Madhya Pradesh, Gwalior, MP 474005, India.

Department of Pharmaceutics, Bharat Institute of Technology (BIT), BIT-School of Pharmacy, Meerut, UP 250103, India.

出版信息

Drug Discov Today. 2022 May;27(5):1431-1440. doi: 10.1016/j.drudis.2022.01.003. Epub 2022 Jan 10.

Abstract

Neurodegenerative disorders (NDs) lead to the progressive degeneration of the structural and physiological functions of the central and peripheral nervous systems, resulting in lifelong cognitive and motor dysfunction. Although comprehensive treatment of NDs is lacking, small interfering (si)RNA has shown therapeutic utility in the form of cellular nuclease-driven downregulation of mRNA levels. Various nanotechnologies have been used to modulate crucial physicochemical and biopharmaceutical properties of siRNA to provide protection and to enhance biomembrane interactions, residence times, tissue absorption, and cellular internalization for improved cytoplasm and/or nucleus interactions. In this review, we highlight advances in, and the role of, siRNA-based nanocarriers for the treatment of various NDs.

摘要

神经退行性疾病(NDs)会导致中枢和外周神经系统的结构与生理功能逐渐退化,从而造成终身的认知和运动功能障碍。尽管目前缺乏针对NDs的综合治疗方法,但小干扰(si)RNA已通过细胞核酸酶驱动下调mRNA水平的方式展现出治疗效用。各种纳米技术已被用于调节siRNA的关键物理化学和生物制药特性,以提供保护并增强生物膜相互作用、延长驻留时间、促进组织吸收以及实现细胞内化,从而改善其与细胞质和/或细胞核的相互作用。在本综述中,我们重点介绍了基于siRNA的纳米载体在治疗各种NDs方面的进展及其作用。

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