Nrf2-Inducers Counteract Neurodegeneration in Frataxin-Silenced Motor Neurons: Disclosing New Therapeutic Targets for Friedreich’s Ataxia

Petrillo, S.; Piermarini, E.; Pastore, A.; Vasco, G.; Schirinzi, T.; Carrozzo, R.; Bertini, E.; Piemonte, F.; Int. J. Mol. Sci. 2017, 18, 2173. doi:10.3390/ijms18102173

In FA, the dys-regulation of cellular antioxidant defenses, due to frataxin deficiency, exacerbates oxidative stress, thus the Nrf2 activation becomes more and more of an attractive strategy for the treatment of this disease.

Overall, our findings support Nrf2 as a therapeutic target for FA, and its induction as a promising approach to prevent or slow the pathological changes observed in this disease. Furthermore, the Nrf2 impairment mirrored at the systemic level in PBMCs of patients may help to open new perspectives for biomarker research in FA, potentially useful for monitoring clinical trials.