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

Derek M Dykxhoorn, Judy Lieberman

Research output: Contribution to journalArticle

273 Citations (Scopus)

Abstract

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
Pages (from-to)401-423
Number of pages23
JournalAnnual Review of Medicine
Volume56
DOIs
StatePublished - Mar 11 2005
Externally publishedYes

Fingerprint

Therapeutic Human Experimentation
RNA Interference
RNA
Genes
Gene expression
RNA-Induced Silencing Complex
Cells
Neurodegenerative diseases
Gene Expression
Animal Diseases
Double-Stranded RNA
Gene Silencing
Sequence Homology
Viruses
Neurodegenerative Diseases
Small Interfering RNA
Animals
Genome
Degradation
Messenger RNA

Keywords

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

ASJC Scopus subject areas

  • Cell Biology
  • Medicine(all)

Cite this

The silent revolution : RNA interference as basic biology, research tool, and therapeutic. / Dykxhoorn, Derek M; Lieberman, Judy.

In: Annual Review of Medicine, Vol. 56, 11.03.2005, p. 401-423.

Research output: Contribution to journalArticle

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