Friedreich Ataxia: current state-of-the-art, and future prospects for mitochondrial-focused therapies
Friedreich ´s Ataxia is an autosomal recessive genetic disease causing the defective gene product, frataxin. A body of literature has been focused on the attempts to counteract frataxin deficiency and the consequent iron imbalance, in order to mitigate the disease-associated prooxidant state and clinical cour se. The present mini review is aimed at evaluating the basic and clinical reports on the roles and the use of a set of iron chelators, antioxidants and some cofactors involved in the key mitochondrial functions.
Source: Translational Research - Category: Research Authors: Federico V. Pallard ó, Giovanni Pagano, Laura R. Rodríguez, Pilar Gonzalez-Cabo, Alex Lyakhovich, Marco Trifuoggi Source Type: research