Treatment with ROS detoxifying gold quantum clusters alleviates the functional decline in a mouse model of Friedreich ataxia

CHIARA VILLA, MARIELLA LEGATO, ALESSANDRO UMBACH, CHIARA RIGANTI, REBECCA JONES, BEATRICE MARTINI, MARINA BOIDO, CLAUDIO MEDANA, IRENE FACCHINETTI, DARIO BARNI, MILENA PINTO, TANIA ARGUELLO, MARZIA BELICCHI, GIGLIOLA FAGIOLARI, CARLA LIACI, MAURIZIO MOGGIO, RICCARDO RUFFO, CARLOS T. MORAES, ANGELO MONGUZZI, GIORGIO R. MERLO, YVAN TORRENTE; Science Translational Medicine 18 Aug 2021: Vol. 13, Issue 607, eabe1633 DOI: 10.1126/scitranslmed.abe1633 

Patients with Friedreich ataxia (FRDA) present neurological deficits and impaired muscle coordination. Mitochondrial energy conversion and oxidative phosphorylation have been shown to contribute to disease pathophysiology. Now, Villa et al. tested whether gold cluster superstructures (Au8-pXs), previously shown to have antioxidant properties, could be effective in reducing the hallmarks of FRDA in vitro and in vivo. Au8-pXs improved mitochondrial function, rescued autophagy flux, and increased FXN protein expression in mesenchymal stem cells from patients. In vivo, the treatment had therapeutic effects in a mouse mode, suggesting that Au8-pXs might be effective in reducing FRDA symptoms.