Investigating the Mechanosensory System in Friedreich’s Ataxia using Stem Cell Models
Discovery of frataxin substitute drug candidates for the treatment of Friedreich’s Ataxia
Trojan nanobodies to increase frataxin function
Measurement of Frataxin mRNA in NHP and human biofluids
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