RNA Interference (RNAi)-Based Therapeutics: Delivering on the Promise?

被引：257
|
作者：
Bobbin, Maggie L. [1 ,2 ]
Rossi, John J. [3 ]
机构：
[1] Massachusetts Gen Hosp, Mol Pathol Unit, Charlestown, MA 02129 USA
[2] Massachusetts Gen Hosp, Ctr Canc Res, Charlestown, MA 02129 USA
[3] City Hope Natl Med Ctr, Beckman Res Inst, Duarte, CA 91010 USA
来源：
ANNUAL REVIEW OF PHARMACOLOGY AND TOXICOLOGY, VOL 56 | 2016年 / 56卷
关键词：
clinical trials; siRNA; miRNA; shRNA; dsiRNA; DOUBLE-STRANDED-RNA; CHRONIC HEPATITIS-B; SIRNA DELIVERY; VIRUS; STRATEGIES; EFFICACY;
D O I：
10.1146/annurev-pharmtox-010715-103633
中图分类号：
R9 [药学];
学科分类号：
1007 ;
摘要：
A resurgence in clinical trials using RNA interference (RNAi) occurred in 2012. Although there were initial difficulties in achieving efficacious results with RNAi without toxic side effects, advances in delivery and improved chemistry made this resurgence possible. More than 20 RNAi-based therapeutics are currently in clinical trials, and several of these are Phase III trials. Continued positive results from these trials have helped bolster further attempts to develop clinically relevant RNAi therapies. With a wide variety of disease targets to choose from, the first RNAi therapeutic to be clinically approved is not far off. This review covers recently established and completed clinical trials.
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收藏
页码：103 / 122
页数：20
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