基于 mRNA 的疗法越来越显示出治疗各种疾病的巨大潜力，包括传染病、癌症和遗传性疾病。有效的递送系统对于推进 mRNA 疗法至关重要。脂质纳米颗粒 (LNP) 作为一种出色的载体，其安全性和耐受性在市售 mRNA 疫苗中得到了广泛验证。标准LNP通常由四种成分组成：可电离脂质（IL）、辅助脂质、胆固醇和聚乙二醇脂质（PEG-脂质），IL的结构设计逐渐成为研究兴趣的焦点。其他成分的化学结构和配方也显着影响 LNP 的递送效率、靶向特异性和稳定性。 LNP 的复杂配方可能会阻碍 mRNA 疗法的临床转化，并引起对其安全性的广泛关注。本综述旨在总结基于 LNPs 的 mRNA 疗法在临床试验中的进展，重点关注这些试验期间发生的不良反应。它还讨论了 LNP 组件的代表性创新，强调了该研究领域的挑战和潜在方法。我们坚信本次审查将促进 LNP 组合物的进一步改进和设计，以优化 mRNA 疗法。本文分类为：生物学的纳米技术方法 > 生物学中的纳米级系统 生物启发的纳米材料 > 基于脂质的结构。© 2024 Wiley periodicals LLC。

mRNA-based therapeutics increasingly demonstrate significant potential in treating various diseases, including infectious diseases, cancers, and genetic disorders. Effective delivery systems are crucial for advancing mRNA therapeutics. Lipid nanoparticles (LNPs) serve as an excellent carrier, widely validated for their safety and tolerability in commercially available mRNA vaccines. Standard LNPs typically consist of four components: ionizable lipids (ILs), helper lipids, cholesterol, and polyethylene glycol-lipids (PEG-lipids), with the structural design of ILs gradually becoming a focal point of research interest. The chemical structures and formulations of the other components also significantly affect the delivery efficiency, targeting specificity, and stability of LNPs. The complex formulations of LNPs may hinder the clinical transformation of mRNA therapeutics and have raised widespread concerns about their safety. This review aims to summarize the progress of LNPs-based mRNA therapeutics in clinical trials, focusing on adverse effects that occurred during these trials. It also discusses representative innovations in LNP components, highlighting challenges and potential ways in this research field. We firmly believe this review will promote further improvements and designs of LNP compositions to optimize mRNA therapeutics. This article is categorized under: Nanotechnology Approaches to Biology > Nanoscale Systems in Biology Biology-Inspired Nanomaterials > Lipid-Based Structures.© 2024 Wiley Periodicals LLC.