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Annu Rev Pharmacol Toxicol. 2016;56:103-22. doi: 10.1146/annurev-pharmtox-010715-103633.

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

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Molecular Pathology Unit and.
Center for Cancer Research, Massachusetts General Hospital, Charlestown, Massachusetts 02129; email:
Beckman Research Institute, City of Hope, Duarte, California 91010; email:


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.


clinical trials; dsiRNA; miRNA; shRNA; siRNA

[Indexed for MEDLINE]

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