Targeting neurological disease with RNAi

Mol Biosyst. 2007 Nov;3(11):773-80. doi: 10.1039/b701169e. Epub 2007 Aug 29.


The neuroscientific community rapidly adopted RNA interference techniques as an experimental tool for the dissection of gene function in vitro and in animal models of neurological disease in vivo. Here, we discuss recent advances in the biotechnical implementation of siRNA/shRNA-mediated gene silencing focusing on issues of design, delivery and putative detrimental effects. We then summarize the current use of RNAi in targeting neurological disease models and give an outlook on the implementation of this technique in clinical therapy.

Publication types

  • Research Support, Non-U.S. Gov't
  • Review

MeSH terms

  • Brain / metabolism
  • Brain / pathology
  • Humans
  • Nervous System Diseases / genetics
  • Nervous System Diseases / therapy*
  • RNA Interference*
  • RNA, Small Interfering / genetics
  • RNA, Small Interfering / metabolism
  • Transfection


  • RNA, Small Interfering