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Article

Epigenetic therapy for <scp>F</scp> riedreich ataxia

27 Aug 2014

Abstract excerpt

OBJECTIVE: To investigate whether a histone deacetylase inhibitor (HDACi) would be effective in an in vitro model for the neurodegenerative disease Friedreich ataxia (FRDA) and to evaluate safety and surrogate markers of efficacy in a phase I clinical trial in patients. METHODS: We used a human FRDA neuronal cell model, derived from patient induced pluripotent stem cells, to determine the efficacy of a...

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PMID: 25159818
DOI: 10.1002/ana.24260

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