Article
Small molecules affecting transcription in Friedreich ataxia.
Pharmacology & therapeutics - 1 Nov 2007
Gottesfeld Joel M
Abstract excerpt
This review concerns the development of small molecule therapeutics for the inherited neurodegenerative disease Friedreich ataxia (FRDA). FRDA is caused by transcriptional repression of the nuclear FXN gene, encoding the essential mitochondrial protein frataxin and accompanying loss of frataxin p...
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