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非病毒载体递送与小干扰 RNA 的治疗应用。

Non-Viral Delivery and Therapeutic Application of Small Interfering RNAs.

机构信息

Engelhardt Institute of Molecular Biology, Russian Academy of Sciences, Vavilova Str., 32, Moscow, Russia, 119991.

出版信息

Acta Naturae. 2013 Jul;5(3):35-53.

Abstract

RNA interference (RNAi) is a powerful method used for gene expression regulation. The increasing knowledge about the molecular mechanism of this phenomenon creates new avenues for the application of the RNAi technology in the treatment of various human diseases. However, delivery of RNA interference mediators, small interfering RNAs (siRNAs), to target cells is a major hurdle. Effective and safe pharmacological use of siRNAs requires carriers that can deliver siRNA to its target site and the development of methods for protection of these fragile molecules from in vivo degradation. This review summarizes various strategies for siRNA delivery, including chemical modification and non-viral approaches, such as the polymer-based, peptide-based, lipid-based techniques, and inorganic nanosystems. The advantages, disadvantages, and prospects for the therapeutic application of these methods are also examined in this paper.

摘要

RNA 干扰 (RNAi) 是一种用于基因表达调控的强大方法。对这一现象分子机制的日益了解为 RNAi 技术在治疗各种人类疾病中的应用开辟了新途径。然而,RNA 干扰介质(小干扰 RNA(siRNA))递送到靶细胞是一个主要的障碍。siRNA 的有效和安全的药理学应用需要能够将 siRNA 递送到其靶位的载体,以及开发保护这些脆弱分子免受体内降解的方法。本文综述了 siRNA 递送的各种策略,包括化学修饰和非病毒方法,如基于聚合物、基于肽、基于脂质的技术和无机纳米系统。本文还研究了这些方法在治疗应用中的优缺点和前景。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/6f3b/3848066/4575f010fb82/AN20758251-18-035-g001.jpg

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