Strategies for Organ-Targeted mRNA Delivery by Lipid Nanoparticles.

Abstract

Messenger RNA (mRNA) technology has rapidly evolved, significantly impacting various therapeutic applications, including vaccines, protein replacement, and gene editing. Lipid nanoparticles (LNPs) have emerged as a pivotal nonviral vector for mRNA delivery, crucial for organ-targeted therapies. Despite their success, most LNP formulations predominantly target the liver, limiting their use in nonliver diseases. This review explores strategies to achieve organ-specific mRNA delivery using LNPs, including the discovery of new lipid structures, modification of targeting ligands, incorporation of additional components, and optimization of LNP formulations. These advancements aim to enhance the precision and efficacy of mRNA therapeutics across a broader range of diseases.