In Vivo Delivery of RNAi with Lipid-Based Nanoparticles

被引：131

作者：

Huang, Leaf ^{[1
]}

Liu, Yang ^{[1
]}

机构：

[1] Univ N Carolina, Div Mol Pharmaceut, Eshelman Sch Pharm, Chapel Hill, NC 27599 USA

来源：

ANNUAL REVIEW OF BIOMEDICAL ENGINEERING, VOL 13 | 2011年 / 13卷

关键词：

RNA interference; siRNA; cationic lipid; gene therapy; SHORT-INTERFERING RNAS; TUMOR-BEARING MICE; PEG IGM PRODUCTION; SIRNA DELIVERY; POLY(ETHYLENE GLYCOL); NONVIRAL VECTORS; MAMMALIAN-CELLS; GENE-EXPRESSION; INTRACELLULAR DELIVERY; CLEAVABLE LIPOPOLYMERS;

D O I：

10.1146/annurev-bioeng-071910-124709

中图分类号：

R318 [生物医学工程];

学科分类号：

0831 ;

摘要：

RNA interference (RNAi) technology represents a fundamentally new category of treatments for human disease by addressing targets that are traditionally considered undruggable with existing medicines. The major challenge for RNAi-based therapy is the delivery system that meets human therapeutic needs. Therefore, engineering vectors with good delivery efficiency and safety profile is an intense area of research. Lipid-based nanoparticles for RNAi have yielded successful advances in vivo and to an extent in clinical trials. In this review, we discuss the barriers in developing lipid-based nanoparticles for in vivo RNAi and different strategies to overcome them. Rational designs that address safety concerns and ensure effective delivery will aid the translation of engineered lipid-based nanoparticles toward the clinic in the foreseeable future.

引用

页码：507 / 530

页数：24

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