体外转录合成mRNA递送材料的研究进展

Research progress in transcription and synthesis of mRNA delivery materials in vitro

信使RNA(mRNA)递送技术是指通过体外转录合成mRNA并递送至细胞内,表达出相应的蛋白进而发挥重要的生物学功能。近年来,mRNA递送技术在疫苗和基因治疗等方面成为研究的前沿和热点。mRNA一般采用纳米粒或非纳米粒材料包裹后进行递送。尽管取得了许多进展,但缺乏有效和无毒的递送材料仍然是限制mRNA应用的主要因素。病毒系统(如慢病毒和腺相关病毒)的使用已被广泛认为是核酸的有效递送方式,但不需要的免疫反应是其发展的根本障碍。因此,越来越多的非病毒载体(包括脂质、聚合物、基于纳米的载体或功能载体)作为安全、有效的递送工具受到关注。该文综述了近年来各种mRNA递送材料的进展、挑战及未来方向。

Messenger RNA(mRNA)delivery technology refers to the synthesis of mRNA by in vitro transcription and delivery to cells,and the expression of corresponding proteins to exert important biological functions.In recent years,mRNA delivery technology has become a research frontier and hotspot in vaccine and gene therapy.mRNA is generally packaged with nanoparticles or non-nanoparticles for delivery.Despite many advances,the lack of effective and non-toxic delivery methods remains a major factor limiting the application of mRNA.The use of viral systems(such as lentiviruses and adeno-associated viruses)has been widely recognized as an effective way to deliver nucleic acids,but an unwanted immune response is a fundamental barrier to its development.As a result,more and more non-viral vectors(including lipids,polymers,nano-based carriers or functional carriers)have attracted attention as safe and effective delivery vehicles.This paper reviewed the progress,challenges and future directions of various mRNA delivery materials in recent years.