The DOI matches its Crossref record; 19 of the 19 references it cites were found in a registry or an index.
Current prospects for RNA interference-based therapies
Open accessAbstract
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.
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Cites
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“For example, using 2′-O-methyl-modified purine nucleosides in the passenger strand reduces IFN induction but retains targeting specificity [38] [39] [40] .”
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Background
Cited by
The HCV Life Cycle: In vitro Tissue Culture Systems and Therapeutic Targets
Gerold, G., & Pietschmann, T. (2014) · Digestive Diseases · doi:10.1159/000360830 · Cited by 4,854
RNA therapeutics: beyond RNA interference and antisense oligonucleotides
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siRNA versus miRNA as therapeutics for gene silencing
Lam, J. K. W., Chow, M. Y. T., Zhang, Y., & Leung, S. W. S. (2015) · Molecular Therapy — Nucleic Acids · doi:10.1038/mtna.2015.23 · Cited by 1,081
Molecular Mechanisms of RNA Interference
Wilson, R. C., & Doudna, J. A. (2013) · Annual Review of Biophysics · doi:10.1146/annurev-biophys-083012-130404 · Cited by 1,076
Cancer nanomedicine for combination cancer immunotherapy
Nam, J., Son, S., Park, K. S., Zou, W., Shea, L. D., & Moon, J. J. (2019) · Nature Reviews Materials · doi:10.1038/s41578-019-0108-1 · Cited by 940
Metadata
- Authors
- Davidson, B. L., & McCray, P. B.
- Venue
- Nature Reviews Genetics
- Published
- 2011-04-18
- Volume
- 12
- Issue
- 5
- Pages
- 329-340
- ISSN
- 1471-0056 (ISSN-L)
- 1471-0064
- OpenAlex
- W1972376076
- Cited by
- 761
- References
- 19
- FWCI
- 6.32
- Type
- journal article
- Language
- en
- Open access
- bronze
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