《生命科学》 2023, 35(8): 1012-1022
核糖核酸(ribonucleic acid, RNA) 疗法通过将外源RNA 引入特定细胞来精准调控基因的表达，从而实现对疾病的干预。近年来，临床结果表明RNA 疗法在基因调控和疾病预防上是一种十分有潜力的基因疗法。然而，RNA 药物在递送过程中仍然面临如稳定性差、组织靶向性弱、免疫原性强等诸多问题，限制了RNA 药物的进一步临床转化应用。本文总结了不同RNA 药物的递送方式在临床前和临床实验中取得的最新研究进展，并讨论不同递送载体的应用前景和局限性，旨在为RNA 药物递送方式的优化提供新的设计思路，促进RNA 疗法的临床应用。
通讯作者：黄承浩 , Email:email@example.com
RNA-based therapies offer a promising approach for treating a variety of diseases by regulating gene expression through introduction of exogenous RNAs into target cells. Clinical data supports the potential of RNA therapeutics in gene regulation and disease prevention. However, RNA-based therapies face several challenges, such as poor stability, weak tissue targeting, and strong immunogenicity during delivery, which have hindered their clinical application. This review highlights recent progress in the development of various RNA drug delivery methods, both in preclinical and clinical trials, then discusses the potential and limitations of different delivery vectors, providing new insights for the optimization of RNA drug delivery methods and the advancement of clinical applications of RNA drugs.
Communication Author：HUANG Cheng-Hao , Email:firstname.lastname@example.org