Defining new epigenetic gene silencing axis driving repeat-associated heterochromatin formation in Friedreich’s Ataxia patients’ cells
Defining and targeting cardiac lipid dysregulation in Friedreich ataxia using human iPSC-derived cardiac models
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
SHIFT-FA – Study of Heterogeneity in Friedreich’s Ataxia Trajectories
Investigating spinal sensory-motor circuit pathology in two mouse models of FA
AIMs_Play: Predicting Disease Progression in Pediatric Friedreich Ataxia with Generative AI-Enabled Platform
Neuro-otology: Diffusion MRI and audiological biomarkers for Friedreich ataxia disease progression
RNA Writing Trans-Splicing for Enhancing Frataxin Translation