RNA therapeutics for Friedreich’s Ataxia

Friedreich's ataxia is a neurodegenerative disease for which no effective cure is currently available. At the basis of the disease there are genetic defects impairing the synthesis of the frataxin protein, whose levels inside cells are significantly reduced. Aim of this project is the development of new tools to raise the intracellular levels of frataxin. If successful, frataxin function will be partially or totally restored and the disease slowed or cured. To reach this goal, we will explore the use of innovative therapeutic strategies based on RNA molecules to rescue pathological defects in patients' cells.