On future's doorstep: RNA interference and the pharmacopeia of tomorrow

J Clin Invest. 2007 Dec;117(12):3612-4. doi: 10.1172/JCI34274.


Small molecules and antibodies have revolutionized the treatment of malignant diseases and appear promising for the treatment of many others. Nonetheless, there are many candidate therapeutic targets that are not amenable to attack by the current generation of targeted therapies, and in a small but growing number of patients, resistance to initially successful treatments evolves. This Review Series on the medicinal promise of posttranscriptional gene silencing with small interfering RNA and other molecules capable of inducing RNA interference (RNAi) is motivated by the hypothesis that effectors of RNAi can be developed into effective drugs for treating malignancies as well as many other types of disease. As this Review Series points out, there is still much to do, but many in the field now hope that the time has finally arrived when "antisense" therapies will finally come of age and fulfill their promise as the magic bullets of the 21st century.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't
  • Review

MeSH terms

  • Gene Expression Regulation, Neoplastic / drug effects*
  • Humans
  • Neoplasms / drug therapy*
  • Neoplasms / genetics
  • Neoplasms / metabolism
  • Pharmacopoeias as Topic
  • RNA Interference / drug effects*
  • RNA Stability*
  • RNA, Small Interfering* / chemistry
  • RNA, Small Interfering* / pharmacokinetics
  • RNA, Small Interfering* / therapeutic use


  • RNA, Small Interfering