Science Spotlight: Dialing in pathway balance in Friedreich ataxia

Two independent groups identified FDX2 reduction as a therapeutic axis for Friedreich ataxia, both publishing their results in Nature. The findings suggest therapeutic strategies should treat FXN as part of a stoichiometric system with FDX2, aiming to normalize the balance between the two proteins.

Mitochondrial iron-sulfur (Fe-S) clusters are cofactors used by many enzymes in crucial biological systems such as the electron transport chain, TCA cycle, DNA repair and translation. Disruption of mitochondrial Fe-S biogenesis causes cells to develop respiratory chain defects, metabolic reprogramming and increased oxidative stress...