amorchem header logo close button Menu
arrow leftBack to portfolio


This program involves using a novel technology (“TALE-FXN”) that has the potential to increase the levels of the Frataxin (“FXN”) protein in a Friedreich’s Ataxia mouse model. This tool is designed to specifically induce transcription of FXN in diseased cells where the gene has been silenced, thereby addressing the root cause of the disease. Activity was demonstrated in cell-based and animal models. This program is led by Dr. Jacques P. Tremblay from the Centre hospitalier universitaire de Québec – Université Laval.