The therapeutic potential of RNA interference

FEBS Lett. 2005 Oct 31;579(26):5996-6007. doi: 10.1016/j.febslet.2005.08.004. Epub 2005 Aug 15.


In recent years, we have witnessed the discovery of a new mechanism of gene regulation called RNA interference (RNAi), which has revitalized interest in the development of nucleic acid-based technologies for therapeutic gene suppression. This review focuses on the potential therapeutic use of RNAi, discussing the theoretical advantages of RNAi-based therapeutics over previous technologies as well as the challenges involved in developing RNAi for clinical use. Also reviewed, are the in vivo proof-of principle experiments that provide the preclinical justification for the continued development of RNAi-based therapeutics.

Publication types

  • Review

MeSH terms

  • Animals
  • Antineoplastic Agents / pharmacology
  • Apoptosis
  • Biotechnology
  • Drug Delivery Systems*
  • Drug Industry
  • Eye Diseases / therapy
  • Gene Expression Regulation*
  • Gene Expression Regulation, Viral
  • Genetic Techniques
  • Humans
  • Inflammation / therapy
  • RNA Interference*
  • RNA, Small Interfering / metabolism
  • RNA, Viral / genetics*


  • Antineoplastic Agents
  • RNA, Small Interfering
  • RNA, Viral