siRNA therapeutics: big potential from small RNAs

Gene Ther. 2005 Jan;12(1):5-11. doi: 10.1038/


RNA interference (RNAi) is now an umbrella term referring to post-transcriptional gene silencing mediated by either degradation or translation arrest of target RNA. This process is initiated by double-stranded RNA with sequence homology driving specificity. The discovery that 21-23 nucleotide RNA duplexes (small-interfering RNAs, siRNAs) mediate RNAi in mammalian cells opened the door to the therapeutic use of siRNAs. While much work remains to optimize delivery and maintain specificity, the therapeutic advantages of siRNAs for treatment of viral infection, dominant disorders, cancer, and neurological disorders show great promise.

Publication types

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

MeSH terms

  • Gene Targeting
  • Genetic Therapy / methods*
  • Humans
  • Neoplasms / therapy*
  • Nervous System Diseases / therapy*
  • RNA Interference
  • RNA, Small Interfering*


  • RNA, Small Interfering