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The current state and future directions of RNAi-based therapeutics

Setten, Ryan L. and Rossi, John J. and Han, Si-ping (2019) The current state and future directions of RNAi-based therapeutics. Nature Reviews. Drug Discovery, 18 (6). pp. 421-446. ISSN 1474-1776.

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The RNA interference (RNAi) pathway regulates mRNA stability and translation in nearly all human cells. Small double-stranded RNA molecules can efficiently trigger RNAi silencing of specific genes, but their therapeutic use has faced numerous challenges involving safety and potency. However, August 2018 marked a new era for the field, with the US Food and Drug Administration approving ONPATTRO, the first RNAi-based drug. In this Review, we discuss key advances in the design and development of RNAi drugs leading up to this landmark achievement, the state of the current clinical pipeline and prospects for future advances, including novel RNAi pathway agents utilizing mechanisms beyond post-translational RNAi silencing.

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Han, Si-ping0000-0002-2213-4953
Additional Information:© 2019 Springer Nature Publishing AG. Published 07 March 2019. This work was funded by US National Institutes of Health grant AI29329 and US National Science Foundation Emerging Frontiers in Research and Innovation (EFRI)–Origami Design for Integration of Self-assembling Systems for Engineering Innovation (ODISSEI) award 133241. Competing interests: J.J.R. is a co-founder of Dicerna Pharmaceuticals and MiNA Therapeutics. S.-p.H. and J.J.R. are inventors on US patents and patent applications for conditional RNA interference-related technologies.
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Subject Keywords:Drug discovery; RNAi therapy
Issue or Number:6
Record Number:CaltechAUTHORS:20190312-140616778
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Usage Policy:No commercial reproduction, distribution, display or performance rights in this work are provided.
ID Code:93740
Deposited By: Tony Diaz
Deposited On:12 Mar 2019 21:13
Last Modified:24 Nov 2020 00:37

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