Modelling Friedreich Ataxia in human iPSC-derived cerebellar organoids
Anatomical and functional analysis of corticospinal tract in FRDA mouse model
Role of the Thromboxane-Prostanoid Receptor in FRDA Cardiomyopathy
Hematopoietic Stem Cell Transplantation in FRDA: Proof-of-concept studies using novel conditioning regimen and the YG8s(GAA)>800 mouse
Elucidation of the peripheral immune axis in FRDA and identification of blood-based biomarkers of inflammation
A tractable vertebrate model that recapitulates key aspects of Friedreich Ataxia
Population and cellular heterogeneity in Friedreich Ataxia
Investigating the effects of frataxin deficiency on the human proteome
Mechanism of neuronal rescue by microglia expressing frataxin and characterization of neuronal defects in Friedreich’s ataxia
AAV-mediated therapy for visual impairment associated with Friedreich’s Ataxia
Uncovering the function of a conserved site in eukaryotic frataxins
Identification of concordant electrophysiological biomarkers in Friedreich Ataxia