A potential therapy for Friedreich's ataxia, a neurodegenerative disease that is caused by gene activation, is described in the October 2006 issue of Nature Chemical Biology.
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.
Joel M Gottesfeld (The Scripps Research Institute, La Jolla, CA, USA)
Abstract available online.
(C) Nature Chemical Biology press release.
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