Defining new epigenetic gene silencing axis driving repeat-associated heterochromatin formation in Friedreich’s Ataxia patients’ cells
|Dec 27 2024
Application of RSwitch technology to improve Friedreich’s ataxia viral gene therapy safety and efficacy: control of FXN transgene expression through a small molecule regulation
|Dec 5 2024
Development of Epigenetic Editors for Frataxin Reactivation
|Dec 19 2023
Modelling Friedreich Ataxia in human iPSC-derived cerebellar organoids
|Dec 20 2022
Advancing novel mitochondrial therapies for FA cardiomyopathy: a pragmatic collaboration to move new therapies forward
|Dec 20 2022
Measuring ataxia in children with Friedreich ataxia
|Dec 20 2022
Imaging of glutathione and GABA in the brain as biomarkers of Friedreich Ataxia
|Dec 19 2022
TGFβ signaling activity as disease biomarkers and therapeutic targets in Friedreich’s Ataxia
|Dec 19 2022
Evaluation of ARMMs-mediated delivery of Cas9 protein complexed with gRNAs as a non-viral disease-modifying strategy for Friedreich’s Ataxia
|Dec 19 2020
Development of autologous transplantation of genetically corrected hematopoietic stem cells for Friedreich Ataxia