Unlocking the Therapeutic Applicability of LNP-mRNA:Chemistry,Formulation,and Clinical Strategies  

作　　者：Xiaonan Huang Yishan Ma Guanghui Ma Yufei Xia 

机构地区：[1]Sinovac Biotech Ltd.,Beijing,PR China [2]State Key Laboratory of Biochemical Engineering,Institute of Process Engineering,Chinese Academy of Sciences,Beijing,PR China [3]School of Chemical Engineering,University of Chinese Academy of Sciences,Beijing,PR China

出　　处：《Research》2025年第1期450-476,共27页研究(英文)

基　　金：supported by the National Key Research and Development Program of China(2022YFE0202500);Distinguished Young Scholars of the National Natural Science Foundation of China(grant no.T2222022);National Key Research and Development Program of China(2021YFC2302402 and 2021YFC2302605);Major Program of the National Natural Science Foundation of China(grant no.T2394503);Key Project of the National Natural Science Foundation of China(grant no.32030062);CAS Project for Young Scientists in Basic Research(YSBR-010);the Foundation for Innovative Research Groups of the National Natural Science Foundation of China(grant no.21821005);Youth Innovation Promotion Association of the Chinese Academy of Sciences(no.2020000053).

摘　　要：Messenger RNA(mRNA)has emerged as an innovative therapeutic modality,offering promising avenues for the prevention and treatment of a variety of diseases.The tremendous success of mRNA vaccines in effectively combatting coronavirus disease 2019(COVID-19)evidences the unlimited medical and therapeutic potential of mRNA technology.Overcoming challenges related to mRNA stability,immunogenicity,and precision targeting has been made possible by recent advancements in lipid nanoparticles(LNPs).This review summarizes state-of-the-art LNP-mRNA-based therapeutics,including their structure,material compositions,design guidelines,and screening principles.Additionally,we highlight current preclinical and clinical trends in LNP-mRNA therapeutics in a broad range of treatments in ophthalmological conditions,cancer immunotherapy,gene editing,and rare-disease medicine.Particular attention is given to the translation and evolution of LNP-mRNA vaccines into a broader spectrum of therapeutics.We explore concerns in the aspects of inadequate extrahepatic targeting efficacy,elevated doses,safety concerns,and challenges of large-scale production procedures.This discussion may offer insights and perspectives on near-and long-term clinical development prospects for LNP-mRNA therapeutics.

关 键 词：therapeutic applicability lipid nanoparticles lnps clinical strategies messenger rna mrna 

分 类 号：R943[医药卫生—药剂学]