Journal article
Current prospects for RNA interference-based therapies
Nature reviews. Genetics, Vol.12(5), pp.329-340
05/2011
DOI: 10.1038/nrg2968
PMCID: PMC7097665
PMID: 21499294
Abstract
RNA interference (RNAi) is a powerful approach for reducing expression of endogenously expressed proteins. It is widely used for biological applications and is being harnessed to silence mRNAs encoding pathogenic proteins for therapy. Various methods - including delivering RNA oligonucleotides and expressing RNAi triggers from viral vectors - have been developed for successful RNAi in cell culture and in vivo. Recently, RNAi-based gene silencing approaches have been demonstrated in humans, and ongoing clinical trials hold promise for treating fatal disorders or providing alternatives to traditional small molecule therapies. Here we describe the broad range of approaches to achieve targeted gene silencing for therapy, discuss important considerations when developing RNAi triggers for use in humans, and review the current status of clinical trials.
Details
- Title: Subtitle
- Current prospects for RNA interference-based therapies
- Creators
- Beverly L Davidson - Department of Internal Medicine, Roy J. and Lucille A. Carver College of Medicine, University of Iowa, Iowa City, Iowa 52242, USA. beverly-davidson@uiowa.eduPaul B McCray Jr
- Resource Type
- Journal article
- Publication Details
- Nature reviews. Genetics, Vol.12(5), pp.329-340
- DOI
- 10.1038/nrg2968
- PMID
- 21499294
- PMCID
- PMC7097665
- ISSN
- 1471-0056
- eISSN
- 1471-0064
- Grant note
- P01 NS050210 / NINDS NIH HHS P01 HL051670 / NHLBI NIH HHS NS50210 / NINDS NIH HHS HL51670 / NHLBI NIH HHS P30 DK054759 / NIDDK NIH HHS DK54759 / NIDDK NIH HHS
- Language
- English
- Date published
- 05/2011
- Academic Unit
- Microbiology and Immunology; Pulmonary Medicine; Stead Family Department of Pediatrics; Internal Medicine
- Record Identifier
- 9984093218802771
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