核酸类治疗方式(诸如信使RNA)具备革命性的潜力，可以改进现代医药并提高现有药物的性能表现。当中的关键挑战在于安全有效地将mRNA输送到目标组织和细胞，并从传递载体中控制其释放。脂质纳米粒（LNP）广泛研究作为药物载体，并被认为是核酸输送领域的最先进技术。本文先介绍mRNA治疗的优势和作用机制，然后讨论基于可离子化脂质设计的LNP平台和mRNA-LNP疫苗在预防传染病及治疗癌症和多种遗传病方面的应用，最后描述mRNA-LNP治疗面临的挑战和未来前景。版权所有 © 2023 Elsevier Inc. 发表。

Nucleic acid-based therapies such as messenger RNA have the potential to revolutionize modern medicine and enhance the performance of existing pharmaceuticals. The key challenges of mRNA-based therapies are delivering the mRNA safely and effectively to the target tissues and cells and controlling its release from the delivery vehicle. Lipid nanoparticles (LNPs) have been widely studied as drug carriers and are considered to be state-of-the-art technology for nucleic acid delivery. In this review, we begin by presenting the advantages and mechanisms of action of mRNA therapeutics. Then we discuss the design of LNP platforms based on ionizable lipids and the applications of mRNA-LNP vaccines for prevention of infectious diseases and for treatment of cancer and various genetic diseases. Finally, we describe the challenges and future prospects of mRNA-LNP therapeutics.Copyright © 2023. Published by Elsevier Inc.