Somatic GAA expansions as a potential therapeutic target for Friedreich ataxia
Maheswaran Kesavan, Massachusetts General Hospital
Manipulation of the Frataxin Expression and Function by CRISPR/Cas9 Genome Editing in Dictyostelium discoideum
Hernán Gentili, iB3-FCEN-Universidad de Buenos Aires
Modeling Friedreich’s ataxia with iPSC-derived brain cells and correction of the defects by gene editing
Priyanka Mishra, University of California, San Diego
Exploring the role of frataxin in the mitochondrial phenotype of Friedreich’s ataxia patients’ cells
Davide Doni, University of Padova
Nicotinamide Riboside improves mitochondrial function in mouse model of Freidreich's Ataxia
Caroline Perry, University of Pennsylvania