Abstract: The invention provides compositions and methods useful for the treatment of Friedreich's ataxia. In some embodiments, the invention provides compositions and methods useful for inhibiting the ubiquitination of frataxin.
Abstract: Described herein are compositions and methods for treating Friedreich's Ataxia (FRDA). In some aspects, mutant forms of frataxin which are resistant to ubiquitination are provided. In some aspects, pharmaceutical compositions comprising mutant frataxin are provided. In further aspects, methods of using mutant frataxin are provided.
Abstract: In one aspect, the invention presents a method of treating Friedreich's ataxia (FRDA) by administering a therapeutically effective amount of an Src inhibitor.