Therapeutic RNA Interference In Neurodegenerative Disease

The RNAi gene regulatory pathway could potentially suppress the expression of a wide range of disease-causing mutant genes. Initial studies of therapeutic RNAi in cell-based and animal models of neurodegenerative disease have produced encouraging results, and human trials are expected to be initiated within the next few years.

Andrew Fire and Craig Mello won the 2006 Nobel Prize in Physiology or Medicine for the discovery of the RNAi pathway. Since their landmark paper was published in 1998, RNAi has become a powerful tool for biological research, enabling virtually any target gene to be suppressed in a sequence-specific manner. In the current article, Gonzalez-Alegre and Paulson highlight the challenges involved in bringing RNAi to the neurology clinic, including safe and efficient delivery of the therapeutic RNAi agent, and the development of reliable measures of treatment efficacy.

Author contact:

Henry L. Paulson (University of Michigan Medical School, Ann Arbor, MI, USA)
E-mail: henry-paulson@uiowa.edu

Editor contact:

Heather Wood (Editor, Nature Clinical Practice Neurology)
E-mail: h.wood@nature.com

Abstract available online.

(C) Nature Clinical Practice Neurology press release.

Message posted by: Trevor M. D'Souza