Lipid Nanoparticles for Nucleic Acid Delivery Beyond the Liver

被引：9

作者：

Saber, Nadine ^{[1
]}

Senti, Mariona Estape ^{[1
]}

Schiffelers, Raymond M. ^{[1
]}

机构：

[1] Univ Med Ctr Utrecht, CDL Res, Heidelberglaan 100, NL-3584 Utrecht, Netherlands

来源：

HUMAN GENE THERAPY | 2024年 / 35卷 / 17-18期

关键词：

nanomedicine; lipid nanoparticle; drug delivery; extrahepatic delivery; gene therapy; nucleic acid; targeted delivery; HEMATOPOIETIC STEM; RNA DELIVERY; CELLS; PEG; THERAPEUTICS; PERMEABILITY; PEGYLATION; ANTIBODIES; SYSTEMS; LIGAND;

D O I：

10.1089/hum.2024.106

中图分类号：

Q81 [生物工程学（生物技术）]; Q93 [微生物学];

学科分类号：

071005 ; 0836 ; 090102 ; 100705 ;

摘要：

Lipid nanoparticles (LNPs) are the most clinically advanced drug delivery system for nucleic acid therapeutics, exemplified by the success of the COVID-19 mRNA vaccines. However, their clinical use is currently limited to hepatic diseases and vaccines due to their tendency to accumulate in the liver upon intravenous administration. To fully leverage their potential, it is essential to understand and address their liver tropism, while also developing strategies to enhance delivery to tissues beyond the liver. Ensuring that these therapeutics reach their target cells while avoiding off-target cells is essential for both their efficacy and safety. There are three potential targeting strategies-passive, active, and endogenous-which can be used individually or in combination to target nonhepatic tissues. In this review, we delve into the recent advancements in LNP engineering for delivering nucleic acid beyond the liver.

引用

页码：617 / 627

页数：11

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