mRNA 脂质纳米粒的开发：靶向与治疗学方面。

Development of mRNA Lipid Nanoparticles: Targeting and Therapeutic Aspects.

机构信息

College of Pharmaceutical Engineering, Shenyang Pharmaceutical University, Shenyang 110016, China.

Center for Chemical Biology and Drug Discovery, Guangzhou Institute of Biomedicine and Health, Chinese Academy of Sciences, Guangzhou 510530, China.

出版信息

Int J Mol Sci. 2024 Sep 22;25(18):10166. doi: 10.3390/ijms251810166.

DOI:10.3390/ijms251810166

PMID:39337651

原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC11432440/

Abstract

Lipid nanoparticles (LNPs) have emerged as leading non-viral carriers for messenger RNA (mRNA) delivery in clinical applications. Overcoming challenges in safe and effective mRNA delivery to target tissues and cells, along with controlling release from the delivery vehicle, remains pivotal in mRNA-based therapies. This review elucidates the structure of LNPs, the mechanism for mRNA delivery, and the targeted delivery of LNPs to various cells and tissues, including leukocytes, T-cells, dendritic cells, Kupffer cells, hepatic endothelial cells, and hepatic and extrahepatic tissues. Here, we discuss the applications of mRNA-LNP vaccines for the prevention of infectious diseases and for the treatment of cancer and various genetic diseases. Although challenges remain in terms of delivery efficiency, specific tissue targeting, toxicity, and storage stability, mRNA-LNP technology holds extensive potential for the treatment of diseases.

摘要

脂质纳米颗粒 (LNPs) 已成为信使 RNA (mRNA) 临床应用中领先的非病毒载体。克服安全有效将 mRNA 递送至靶组织和细胞的挑战，以及控制从递药载体的释放，对于基于 mRNA 的治疗仍然至关重要。本文阐述了 LNPs 的结构、mRNA 递药机制以及 LNPs 向各种细胞和组织的靶向递送，包括白细胞、T 细胞、树突状细胞、库普弗细胞、肝内皮细胞以及肝内和肝外组织。在此，我们讨论了 mRNA-LNP 疫苗在预防传染病以及治疗癌症和各种遗传疾病中的应用。尽管在递送效率、特定组织靶向性、毒性和储存稳定性方面仍存在挑战，但 mRNA-LNP 技术在疾病治疗方面具有广泛的潜力。