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
Friedreich ataxia (FRDA) is caused by the reduced expression of the mitochondrial protein frataxin (FXN) due to an intronic GAA trinucleotide repeat expansion in the FXN gene. Although FRDA has no cure and few treatment options, there is research dedicated to finding an agent that can curb disease progression and address symptoms as neurobehavioral deficits, muscle endurance, and heart contractile dysfunctions. Because oxidative stress and mitochondrial dysfunctions are implicated in FRDA, we demonstrated the systemic delivery of catalysts activity of gold cluster superstructures (Au8 -pXs) to improve cell response to mitochondrial reactive oxygen species and thereby alleviate FRDA-related pathology in mesenchymal stem cells from patients with FRDA...
August 18, 2021: Science Translational Medicine