RNA interference in the clinic: challenges and future directions

Nat Rev Cancer. 2011 Jan;11(1):59-67. doi: 10.1038/nrc2966. Epub 2010 Dec 16.


Inherent difficulties with blocking many desirable targets using conventional approaches have prompted many to consider using RNA interference (RNAi) as a therapeutic approach. Although exploitation of RNAi has immense potential as a cancer therapeutic, many physiological obstacles stand in the way of successful and efficient delivery. This Review explores current challenges to the development of synthetic RNAi-based therapies and considers new approaches to circumvent biological barriers, to avoid intolerable side effects and to achieve controlled and sustained release.

Publication types

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

MeSH terms

  • Clinical Trials as Topic
  • Humans
  • Neoplasms / genetics
  • Neoplasms / therapy*
  • RNA Interference / physiology*