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RNA药物非病毒递送系统研究进展
引用本文:陈欢, 魏利军. RNA药物非病毒递送系统研究进展[J]. 药学进展, 2022, 46(11): 839-847. DOI: 10.20053/j.issn1001-5094.2022.11.005
作者姓名:陈欢  魏利军
作者单位:1.哈药集团股份有限公司产品立项部 北京 100023
摘    要:
RNA疗法通过将外源性的RNA引入特定细胞来调控基因表达,是一种非常有潜力的疾病治疗策略。然而,由于存在体内稳定性差、难以高效进入靶细胞等问题,RNA药物的递送需借助合适的药物递送系统。与病毒载体相比,非病毒载体具有更高的安全性,已成为本领域的研究热点。主要介绍脂质纳米颗粒递送系统、生物偶联递送系统以及外泌体递送系统这3种可帮助RNA疗法进入临床的非病毒载体递送系统,并对其技术进展和难点进行分析与总结。

关 键 词:RNA药物  非病毒递送系统  脂质纳米粒  生物偶联  外泌体
收稿时间:2022-09-22

Advances in Research on Non-viral Delivery Systems for RNA Drugs
CHEN Huan, WEI Lijun. Advances in Research on Non-viral Delivery Systems for RNA Drugs[J]. Progress in Pharmaceutical Sciences, 2022, 46(11): 839-847. DOI: 10.20053/j.issn1001-5094.2022.11.005
Authors:CHEN Huan  WEI Lijun
Affiliation:1.Product Portfolio Department, Harbin Pharmaceutical Group Co., Ltd., Beijing 100023, China
Abstract:
RNA therapy, which regulates gene expression by introducing exogenous RNAs into specific cells, is a very promising strategy for disease treatment. However, the delivery of RNA drugs require suitable drug delivery systems, because RNAs are unstable in vivo and cannot enter target cells efficiently. Compared with viral vectors, non-viral vectors are safer, and have become a research hotspot. In this article, we mainly introduced three non-viral delivery systems that may enable the clinical translation of RNA therapeutics:lipid nanoparticle delivery systems, bioconjugation delivery systems, and exosome delivery systems. The related technical progress and difficulties were also analyzed and summarized.
Keywords:RNA drug  non-viral delivery system  lipid nanoparticle  bioconjugation  exosome
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