Chemical modification: the key to clinical application of RNA interference?

J Clin Invest. 2007 Dec;117(12):3615-22. doi: 10.1172/JCI33483.


RNA interference provides a potent and specific method for controlling gene expression in human cells. To translate this potential into a broad new family of therapeutics, it is necessary to optimize the efficacy of the RNA-based drugs. As discussed in this Review, it might be possible to achieve this optimization using chemical modifications that improve their in vivo stability, cellular delivery, biodistribution, pharmacokinetics, potency, and specificity.

Publication types

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

MeSH terms

  • Gene Expression Regulation / drug effects*
  • Humans
  • RNA Interference / drug effects*
  • RNA Stability*
  • RNA, Small Interfering* / chemistry
  • RNA, Small Interfering* / pharmacology
  • RNA, Small Interfering* / therapeutic use


  • RNA, Small Interfering