Strategies for silencing human disease using RNA interference
Abstract
Since the first description of RNA interference (RNAi) in animals less than a decade ago, there has been rapid progress towards its use as a therapeutic modality against human diseases. Advances in our understanding of the mechanisms of RNAi and studies of RNAi in vivo indicate that RNAi-based therapies might soon provide a powerful new arsenal against pathogens and diseases for which treatment options are currently limited. Recent findings have highlighted both promise and challenges in using RNAi for therapeutic applications. Design and delivery strategies for RNAi effector molecules must be carefully considered to address safety concerns and to ensure effective, successful treatment of human diseases.
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RNA interference triggers degradation of particular RNA targets by double-stranded RNAs, resulting in down-regulation of corresponding proteins.
“RNA interference (RNAi) is an endogenous cellular process that triggers the degradation of particular RNA targets by double-stranded RNAs, result in the subsequent down-regulation of corresponding proteins.565–567”
From Recent advances in targeting the “undruggable” proteins: from drug discovery to clinical trials (2023) · cites this paper as [566]
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Suzuki, K., Juelich, T., Lim, H., Ishida, T., Watanebe, T., Cooper, D. A., Rao, S., & Kelleher, A. D. (2008) · Journal of Biological Chemistry · doi:10.1074/jbc.m709651200 · Cited by 108 · see citing works
- Authors
- Kim, D. H., & Rossi, J. J.
- Affiliations
- Beckman Research Institute (US)
- Corresponding author
- Rossi, John J.
- ORCID
- Rossi, John J.
- Venue
- Nature Reviews Genetics
- Published
- 2007-03
- Volume
- 8
- Issue
- 3
- Pages
- 173-184
- ISSN
- 1471-0056 (ISSN-L)
- 1471-0064
- OpenAlex
- W1976060898
- Cited by
- 1,078
- References
- 92
- Citations by year
- 2026: 14 · 2025: 22 · 2024: 28 · 2023: 24 · 2022: 33 · 2021: 26 · 2020: 31 · 2019: 41 · 2018: 44 · 2017: 39 · 2016: 38 · 2015: 79 · 2014: 59 · 2013: 85 · 2012: 85
- FWCI
- 8.74
- Type
- journal article
- Language
- en
- Open access
- closed
- Licence
- www.springer.com/tdm (tdm)
- Locations
- Nature Reviews Genetics (publishedVersion)
- PubMed (publishedVersion)
- Funders
- National Institutes of Health
- Topics
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › RNA Interference and Gene Delivery
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › RNA regulation and disease
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › RNA Research and Splicing
- Keywords
- RNA interference, RNAi therapeutics, gene silencing, RNAi delivery, RNAi mechanism
- MeSH terms
- Animals, Eye Diseases, Humans, Neoplasms, Virus Diseases, Genetic Therapy, Neurodegenerative Diseases, RNA Interference, RNA, Small Interfering
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