The promises and pitfalls of RNA-interference-based therapeutics

Nature. 2009 Jan 22;457(7228):426-33. doi: 10.1038/nature07758.


The discovery that gene expression can be controlled by the Watson-Crick base-pairing of small RNAs with messenger RNAs containing complementary sequence - a process known as RNA interference - has markedly advanced our understanding of eukaryotic gene regulation and function. The ability of short RNA sequences to modulate gene expression has provided a powerful tool with which to study gene function and is set to revolutionize the treatment of disease. Remarkably, despite being just one decade from its discovery, the phenomenon is already being used therapeutically in human clinical trials, and biotechnology companies that focus on RNA-interference-based therapeutics are already publicly traded.

Publication types

  • Research Support, N.I.H., Extramural
  • Review

MeSH terms

  • Animals
  • Clinical Trials as Topic
  • Gene Knockdown Techniques*
  • Genetic Therapy / adverse effects
  • Genetic Therapy / trends*
  • Humans
  • RNA Interference*
  • RNA, Small Interfering / administration & dosage
  • RNA, Small Interfering / adverse effects
  • RNA, Small Interfering / genetics
  • RNA, Small Interfering / therapeutic use*


  • RNA, Small Interfering