Epigenetic-based therapies for Friedreich ataxia.
London, United Kingdom. In Front Genet, 2013
Friedreich ataxia (FRDA) is a lethal autosomal recessive neurodegenerative disorder caused primarily by a homozygous GAA repeat expansion mutation within the first intron of the FXN gene, leading to inhibition of FXN transcription and thus reduced frataxin protein expression.
miR-886-3p levels are elevated in Friedreich ataxia.
New York City, United States. In J Neurosci, 2012
Selective reduction in microRNA (miR)-886-3p by an anti-miR leads to elevation of FXN message and protein levels without associated changes in histone marks at the FXN locus.