Interfering with disease: opportunities and roadblocks to harnessing RNA interference

Trends Mol Med. 2003 Sep;9(9):397-403. doi: 10.1016/s1471-4914(03)00143-6.


RNA interference (RNAi) is an evolutionarily conserved mechanism for silencing gene expression by targeted degradation of mRNA. Short double-stranded RNAs, known as small interfering RNAs (siRNA), are incorporated into an RNA-induced silencing complex that directs degradation of RNA containing a homologous sequence. RNAi has been shown to work in mammalian cells, and can inhibit viral infection and control tumor cell growth in vitro. Recently, it has been shown that intravenous injection of siRNA or of plasmids expressing sequences processed to siRNA can protect mice from autoimmune and viral hepatitis. RNAi could provide an exciting new therapeutic modality for treating infection, cancer, neurodegenerative disease and other illnesses.

Publication types

  • Review

MeSH terms

  • Animals
  • Genetic Therapy / adverse effects
  • Genetic Therapy / methods*
  • Genetic Therapy / trends*
  • Genetic Vectors / genetics
  • 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