Xu Sijia, Hu Zhenzhen, Song Fenglin, Xu Ying, Han Xuexiang
Key Laboratory of RNA Innovation, Science and Engineering, CAS Center for Excellence in Molecular Cell Science, Shanghai Institute of Biochemistry and Cell Biology, Chinese Academy of Sciences, University of Chinese Academy of Sciences, Shanghai 200031, China.
School of Pharmacy, East China Normal University, Shanghai 200241, China.
Mol Ther Methods Clin Dev. 2025 Apr 8;33(2):101463. doi: 10.1016/j.omtm.2025.101463. eCollection 2025 Jun 12.
Lipid nanoparticles (LNPs) are lead non-viral vectors for delivering nucleic acids. LNPs can efficiently encapsulate nucleic acids, protect them from degradation, enhance cellular uptake and induce endosome escape, which show high transfection efficiency and low immunogenicity. In this review, we first introduce the LNP components, highlighting their critical roles in encapsulation, stability, delivery efficiency, and tissue tropism. Next, we summarize different techniques for LNP formulation with a focus on their advantages and disadvantages. Then, we discuss the diverse applications of LNPs in preclinical and clinical studies. Finally, we provide perspectives in the future development of LNPs.
脂质纳米颗粒（LNPs）是用于递送核酸的主要非病毒载体。LNPs能够有效地封装核酸，保护其不被降解，增强细胞摄取并诱导内体逃逸，显示出高转染效率和低免疫原性。在本综述中，我们首先介绍LNP的组成部分，强调它们在封装、稳定性、递送效率和组织嗜性方面的关键作用。接下来，我们总结了LNP制剂的不同技术，重点介绍其优缺点。然后，我们讨论了LNPs在临床前和临床研究中的各种应用。最后，我们对LNPs的未来发展提供了展望。
