#IARC2017 – Newly Potent Small Molecules Help Frataxin to Build Up in Cells, FA Study Finds

A new set of small molecules specifically targeting the ubiquitin degradation system inside cells was found to effectively increase frataxin protein levels in several cell types taken from Friedreich’s ataxia (FA) patients, and merits further investigation. The study reporting these findings, “Highly specific ubiquitin-competing molecules promote frataxin accumulation in Friedreich ataxia iPSC-derived neuronal…