The role of lipid components in lipid nanoparticles for vaccines and gene therapy

Adv Drug Deliv Rev. 2022 Jul 1:114416. doi: 10.1016/j.addr.2022.114416. Online ahead of print.ABSTRACTLipid nanoparticles (LNPs) play a key role in mRNA vaccines against COVID-19. In addition, many preclinical and clinical studies, including the siRNA-LNP product, Onpattro®, highlight that LNPs unlock the potential of nucleic acid-based therapies and vaccines. To understand how this 'key' works, we need to learn about the building blocks that constitute LNPs. In this review, we discuss what each lipid component adds to the LNP delivery platform in terms of size, structure, stability, apparent pKa, nucleic acid encapsulation efficiency, cellular uptake, and endosomal escape. To explore this, we present findings from the liposome field and from landmark and recent articles in the LNP literature. We also discuss challenges and strategies related to in vitro/in vivo studies of LNPs based on fluorescence readouts, immunogenicity/reactogenicity, and LNP delivery beyond the liver. How these fundamental challenges are pursued, including what lipid components are added and combined, will likely determine the scope of LNP-based gene therapies and vaccines for treating various diseases.PMID:35787388 | PMC:PMC9250827 | DOI:10.1016/j.addr.2022.114416
Source: Advanced Drug Delivery Reviews - Category: Drugs & Pharmacology Authors: Source Type: research