Potential application of RNAi for understanding and therapy of neurodegenerative diseases

Front Biosci (Landmark Ed). 2009 Jan 1;14:297-320. doi: 10.2741/3245.


RNA interference (RNAi) technique has become a valuable tool in biology and biomedicine. In the future, it has the potential for application in many different fields including clinical medicine and agriculture. RNAi is a revolution in biology, representing the natural biological process in which genes are turned off in cells, and a completely new approach to drug discovery and development. This review focuses on the therapeutic potential of RNAi for central nervous system diseases. It gives an overview of the progress which has been made in this field to date, including the application of RNAi in vitro (to neurons) and in vivo (animal disease models) and addresses challenges in developing RNAi-based therapies. This review attempts to describe the future prospective of the clinical application of RNAi for neurodegenerative disorders.

Publication types

  • Review

MeSH terms

  • Animals
  • Humans
  • Neurodegenerative Diseases / genetics
  • Neurodegenerative Diseases / therapy*
  • RNA Interference*