体外转录mRNA药物的技术进展和应用前景  

In vitro transcribed(IVT)mRNA drugs:technical progress and application prospect

作　　者：侯书婷 于传飞[1] 王兰[1] 王军志[1] HOU Shu-ting;YU Chuan-fei;WANG Lan;WANG Jun-zhi(National Institutes for Food and Drug Control,Beijing 102629,China)

机构地区：[1]中国食品药品检定研究院,北京102629

出　　处：《药学学报》2023年第8期2047-2058,共12页Acta Pharmaceutica Sinica

基　　金：2022年度国家药品标准制修订研究课题(2022S04)。

摘　　要：体外转录(in vitro transcribed,IVT)mRNA技术发展迅速,COVID-19大流行中mRNA疫苗的广泛应用引起了公众对mRNA技术的讨论。相比传统蛋白药物,IVT mRNA有成本低、模块化生产以及序列易调整等优势,具有广泛的应用前景;但由于mRNA药物的历史较短,其缺乏足够的临床数据,且除mRNA疫苗以外的mRNA药物没有质量控制标准。本文讨论了IVT mRNA药物的序列设计、递送载体、给药方式、应用前景及安全性,并对其质量控制进行简单探讨。The in vitro transcribed(IVT)mRNA technology has progressed rapidly and the application of mRNA vaccines in the COVID-19 pandemic made it become the most talked-about topic.Compared with protein drugs,IVT mRNA has a lower cost;it can be modular produced and its sequence can be modified easily,so it has a broad application prospect.However,due to its short history,mRNA drugs face the problem of lacking sufficient clinical data,and there is no quality control standard for mRNA drugs except mRNA vaccines.We overview the sequence design,delivery vectors,administration,application prospect and safety considerations of mRNA drugs.We also discussed the quality control of mRNA drugs briefly.

关 键 词：体外转录mRNA技术 mRNA序列设计 mRNA递送 mRNA药物 质量控制 

分 类 号：R917[医药卫生—药物分析学]