Published June 2019
| public
Journal Article
The current state and future directions of RNAi-based therapeutics
- Creators
- Setten, Ryan L.
- Rossi, John J.
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Han, Si-ping
Abstract
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.
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.Additional details
- Eprint ID
- 93740
- Resolver ID
- CaltechAUTHORS:20190312-140616778
- NIH
- AI29329
- NSF
- EFMA-1332411
- Created
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2019-03-12Created from EPrint's datestamp field
- Updated
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2023-06-01Created from EPrint's last_modified field