Inhibition of the TLR4 receptor prevents neuro-inflammation and degeneration in frataxin-deficient sensory neurons
Characterization of ocular phenotype of the YG8-800 mouse model of FA and retinal-targeted treatment by AAV based CRISPR-Cas9 gene editing
Unraveling Epigenetic Mechanisms and Therapeutic Strategies for FXN Silencing in Friedreich’s Ataxia
Paired Prime Editors to treat Friedreich’s Ataxia
Treatment of Friedreich’s ataxia using novel ferroptosis inhibitors
Developing and delivering of novel gene editing technologies to contract GAA repeats in Friedreich’s ataxia
An optimized deep learning-based method for cerebellum segmentation
Mechanism of repeat expansion in Friedreich’s ataxia
An induced pluripotent stem cell model to investigate microglia in Friedreich Ataxia
Repeat associated non-AUG (RAN) proteins in FA: contribution to disease and therapeutic opportunities
LEOPARD-FA: Longitudinal Endpoint Optimization to Provide an Assessment of Relevant Drugs in Friedreich’s Ataxia
Understanding ketogenesis in FRDA: Pathophysiology, biomarkers and nutritional therapies