New Compounds Give Voice To Silenced Neural DNA

Frataxin, a protein that protects neurons from cell death caused by free radicals, is 'silenced' in the neurodegenerative disease Friedreich's ataxia. The production of a gene can be regulated by changing the chemical tags attached to nearby histones, or beads that DNA is wrapped around inside the cell. In Friedreich's ataxia, an expansion in the 'noncoding' portion of FXN (the gene encoding frataxin) and modification of the gene's associated histones results in silencing FXN transcription. Gottesfeld and colleagues have identified a class of inhibitors that reverse the silencing in cells in people with Friedreich's ataxia. The inhibitors specifically changed the chemical modifications of the histones around FXN.

These compounds now join the handful of therapeutics that activate specific gene transcription in a human disease.

Author contact:

Joel M Gottesfeld (The Scripps Research Institute, La Jolla, CA, USA)
E-mail: joelg@scripps.edu

Abstract available online.

(C) Nature Chemical Biology press release.

Message posted by: Trevor M. D'Souza