Knocking down barriers: advances in siRNA delivery

被引：2443

作者：

Whitehead, Kathryn A. ^{[2
]}

Langer, Robert ^{[1
,2
]}

Anderson, Daniel G. ^{[1
]}

机构：

[1] MIT, David H Koch Inst Integrated Canc Res, Cambridge, MA 02142 USA

[2] MIT, Dept Chem Engn, Cambridge, MA 02142 USA

来源：

NATURE REVIEWS DRUG DISCOVERY | 2009年 / 8卷 / 02期

关键词：

SHORT INTERFERING RNA; IN-VIVO DELIVERY; MEDIATED DNA-TRANSFECTION; GENE DELIVERY; EFFICIENT DELIVERY; NONVIRAL DELIVERY; COMPLEX MICELLES; HIGHLY EFFICIENT; CATIONIC LIPIDS; PROTECTS MICE;

D O I：

10.1038/nrd2742

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：

In the 10 years that have passed since the Nobel prize-winning discovery of RNA interference (RNAi), billions of dollars have been invested in the therapeutic application of gene silencing in humans. Today, there are promising data from ongoing clinical trials for the treatment of age-related macular degeneration and respiratory syncytial virus. Despite these early successes, however, the widespread use of RNAi therapeutics for disease prevention and treatment requires the development of clinically suitable, safe and effective drug delivery vehicles. Here, we provide an update on the progress of RNAi therapeutics and highlight novel synthetic materials for the encapsulation and intracellular delivery of nucleic acids.

引用

页码：129 / 138

页数：10

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