Unlocking the Potential of the Human Genome With RNA Interference

Nature. 2004 Sep 16;431(7006):371-8. doi: 10.1038/nature02870.


The discovery of RNA interference (RNAi) may well be one of the transforming events in biology in the past decade. RNAi can result in gene silencing or even in the expulsion of sequences from the genome. Harnessed as an experimental tool, RNAi has revolutionized approaches to decoding gene function. It also has the potential to be exploited therapeutically, and clinical trials to test this possibility are already being planned.

Publication types

  • Research Support, U.S. Gov't, Non-P.H.S.
  • Research Support, U.S. Gov't, P.H.S.
  • Review

MeSH terms

  • Animals
  • Drug Evaluation, Preclinical / methods
  • Genetic Therapy / methods*
  • Genome, Human*
  • Genomics / methods*
  • Humans
  • RNA Interference*
  • RNA, Small Interfering / genetics
  • RNA, Small Interfering / metabolism


  • RNA, Small Interfering