mRNA Therapy:Past,Present and Future
10.13865/j.cnki.cjbmb.2024.12.1287
- VernacularTitle:mRNA疗法:过去、现在和未来
- Author:
Meng-Ze SUN
1
;
Peng-Cui LI
;
Xiao-Qing HU
Author Information
1. 北京大学第三医院运动医学科,北京大学运动医学研究所,运动医学关节伤病北京市重点实验室,北京 100191
- Publication Type:Journal Article
- Keywords:
messenger RNA(mRNA);
mRNA delivery;
mRNA therapy
- From:
Chinese Journal of Biochemistry and Molecular Biology
2025;41(2):178-189
- CountryChina
- Language:Chinese
-
Abstract:
mRNA therapy involves delivering target molecules in the form of mRNA into cells to treat dis-eases.The highly variable nature of mRNA sequences offers potential solutions for high-throughput drug discovery and personalized treatment.This review begins with an overview of the development history of mRNA,tracing its journey from discovery to becoming a potential treatment.The review also discusses the applications of mRNA in protein replacement therapy,cancer treatment,in vivo gene editing,and in-fectious disease prevention based on the different categories of proteins delivered by mRNA.Additionally,optimizing mRNA formulations and their delivery vehicles is crucial for clinical application.This review further explains how to enhance the translation efficiency and stability of mRNA through nucleoside modi-fications and sequence optimization,and we systematically compare the pros and cons of novel circular mRNA versus traditional linear mRNA in vaccine development.Moreover,we summarize common deliv-ery methods,such as lipid nanoparticles,and discuss the latest advancements in targeted delivery sys-tems.For currently approved and in-development mRNA drugs,we systematically review the diseases treated,effector molecules delivered,and their clinical stages.Finally,we explore the challenges facing mRNA therapies and the potential diseases they could address,aiming to provide a theoretical foundation and reference for the development of mRNA therapies.
