Friedreich ataxia is a genetic disorder caused by a GAA expansion in intron 1 of the FXN gene, which encodes frataxin. In two recently published studies, the pathological consequences of this FXN mutation have been successfully counteracted in in vitro and in vivo models with the use of different approaches,…
http://www.nature.com/nrneurol/journal/vaop/ncurrent/full/nrneurol.2016.19.html