The silent revolution: RNA interference as basic biology, research tool, and therapeutic

Derek M. Dykxhoorn, Judy Lieberman

Research output: Contribution to journalReview articlepeer-review

279 Scopus citations


RNA interference (RNAi) is an evolutionarily conserved mechanism for silencing gene expression. In primitive organisms, RNAi protects the genome from viruses and other insertable genetic elements and regulates gene expression during development. The antisense (guide) strand of short double-stranded RNAs is incorporated into an RNA-induced silencing complex that can either suppress protein expression or direct degradation of messenger RNAs that contain homologous sequence(s). The discovery that RNAi works in mammalian cells has sparked intense investigation into its role in normal mammalian cell function, its use as a tool to understand or screen for genes functioning in cellular pathways in healthy and diseased cells and animals, and its potential for therapeutic gene silencing. RNAi may provide an important new therapeutic modality for treating infection, cancer, neurodegenerative disease, and other illnesses, although in vivo delivery of small interfering RNAs into cells remains a significant obstacle.

Original languageEnglish (US)
Pages (from-to)401-423
Number of pages23
JournalAnnual review of medicine
StatePublished - 2005
Externally publishedYes


  • miRNA
  • Posttranscriptional gene silencing gene therapy
  • Small interfering RNA

ASJC Scopus subject areas

  • Cell Biology
  • Medicine(all)


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