Molecular medicine for the brain: silencing of disease genes with RNA interference

Lancet Neurol. 2004 Mar;3(3):145-9. doi: 10.1016/S1474-4422(04)00678-7.


The recent discovery of RNA interference (RNAi) has revolutionised biological research and now holds promise as a potential therapy for human diseases. Currently untreatable neurological diseases are especially attractive targets. Scientists have already succeeded in using RNAi to suppress dominant disease genes in vitro; in some cases, this suppression has been allele-specific, silencing the disease-causing allele while maintaining expression of the normal allele. The challenge now is to bring this powerful technology in vivo to animal models to suppress disease genes and correct disease phenotypes. In the confrontation of this challenge, research should benefit from recent advances in viral and non-viral delivery of therapy to the brain.

Publication types

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

MeSH terms

  • Animals
  • Brain Diseases / genetics
  • Brain Diseases / physiopathology
  • Brain Diseases / therapy*
  • Gene Expression Regulation / genetics
  • Gene Silencing / physiology*
  • Gene Transfer Techniques / trends
  • Genetic Therapy / methods*
  • Genetic Therapy / trends*
  • Genetic Vectors / genetics
  • Genetic Vectors / therapeutic use
  • Humans
  • RNA Interference / physiology*
  • RNA, Small Interfering / genetics
  • RNA, Small Interfering / therapeutic use


  • RNA, Small Interfering