Tunable genetic circuits to enable precision genetic medicines for Friedreich’s Ataxia
CBM-01, A Novel Inhibitor of Lipid Peroxidation for Targeting Iron-mediated Pathology in FA Cardiomyopathy
Investigating molecular mechanisms underlying microglia hyperactivation in Friedreich Ataxia
Activation of the ZBP1–cGAS–STING–Type I Interferon Axis as a Mechanistic Driver of Cardiomyopathy in Friedreich’s Ataxia
Investigating Synthetic Gene Regulator function in FRDA patient-derived Cardiomyocytes and neuronal cell types
Does Frataxin Deficiency Disrupt GluR2 Trafficking in the Cerebellum via Defects in Palmitoylation?
Leveraging Real-World Data to Define Clinical Milestones and Assess Pharmacologic Impact in Friedreich’s Ataxia Progression
Preclinical evaluation of novel targets for FRDA cardiomyopathy
Lipidomic profiling and analyses of Friedreich’s Ataxia iPSC-derived dorsal root ganglia sensory neurons
Shortening GAA repeats in Friedreich’s Ataxia
NRF2 and MAO-Ai therapy to improve cardiac health and prevent arrhythmias in Friedreich’s ataxia
Postnatal development of the cerebellar circuitry in the KIKO mouse model of Friedreich’s ataxia