Development of a potential therapy for Friedreich ataxia based on transduction of the frataxin protein in the mitochondria

Canadian Association for Familial Ataxias - Claude St-Jean Foundation
August 25, 2010,

CAFA IS LAUNCHING A MAJOR RESEARCH PROJECT

This research project’s goal is to develop a therapy for Friedreich ataxia by targeting the actual cause of the illness, the reduction of frataxin. The project will therefore aim to administer the frataxin protein intravenously. However, as this protein does not spontaneously penetrate cells, it will be encapsulated with peptides (fragments of other proteins), in nanoparticles. Alternatively, the frataxin protein itself will be modified by adding peptides which will allow the proteins to penetrate not only the interior of cells, but also the interior of the mitochondria.