Cited by 1,078RNA Interference and Gene DeliveryRNA regulation and diseaseRNA Research and Splicing

Strategies for silencing human disease using RNA interference

Kim, Daniel H., Rossi, John J.·2007·Nature Reviews Genetics

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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Crossref · 10.1038/nrg2006

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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”

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Authors
Kim, D. H., & Rossi, J. J.
Affiliations
Beckman Research Institute (US)
Corresponding author
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
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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