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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 protein. Frataxin insufficiency leads to mitochrondrial dysfunction and progressive neurodegeneration, along with...

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Source

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Identifiers

PMID: 17826840
DOI: 10.1016/j.pharmthera.2007.06.014
PMCID: PMC2080619

Genes

FXN
GAA

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-08.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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