Maintaining the silence: reflections on long-term RNAi

Drug Discov Today. 2008 Nov;13(21-22):917-31. doi: 10.1016/j.drudis.2008.06.008. Epub 2008 Aug 7.

Abstract

Since the demonstration of RNA interference (RNAi) in mammalian cells, considerable research and financial effort has gone towards implementing RNAi as a viable therapeutic platform. RNAi is, without doubt, the most promising strategy for the treatment of human genetic disorders. Because many of the targets proposed for RNAi therapy require chronic treatment, researchers agree that the emphasis must now be placed on the safe and long-term application of RNAi drugs to reap the benefits at last.

Publication types

  • Review

MeSH terms

  • Animals
  • Gene Silencing*
  • Humans
  • Kinetics
  • Models, Statistical
  • RNA Interference
  • RNA, Small Interfering / administration & dosage
  • RNA, Small Interfering / adverse effects
  • RNA, Small Interfering / therapeutic use*

Substances

  • RNA, Small Interfering