创新链/学科链/研发链/产业链

新药研发前沿动态 / 医药领域趋势进展

RNA药物非病毒递送系统研究进展

Advances in Research on Non-viral Delivery Systems for RNA Drugs

  • 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。

     

    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.

     

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