Restoring frataxin production remains a central goal of therapy development. This project will investigate a novel class of Synthetic Genome Regulators (SynGRs) designed to target the expanded GAA repeats that silence the FXN gene in most individuals with Friedreich ataxia.
First-in-class SynGR (SynTEF1/SynGR1) compounds have already shown the ability to increase frataxin expression in patient-derived cells, including heart cells and neurons. Researchers will investigate how SynGR1 and next-generation SynGRs (SynGR1 and Next-generation SynGRs) reactivate the FXN gene, determine their effects on gene expression across the genome, and evaluate whether they can restore mitochondrial function in affected cells.
The findings will provide important insights into how these synthetic regulators work and help guide the development of next-generation therapies aimed at restoring frataxin production. This work could advance a promising new approach for addressing the underlying cause of Friedreich ataxia.