This project explores innovative therapies for Friedreich’s ataxia (FRDA), a neurodegenerative disease caused by the silencing of the FXN gene, leading to frataxin deficiency.
Three therapeutic strategies are analyzed: epigenetic reactivation (unlocking the FXN gene using HDAC inhibitors), mRNA therapy (delivering synthetic frataxin mRNA via nanoparticles), and stem cell gene therapy (genetically modifying patient-derived stem cells to produce frataxin).
Atomic Force Microscopy (AFM) is used to assess chromatin structure, mitochondrial function, and cellular mechanics to evaluate treatment efficacy. The study aims to optimize delivery methods, establish biophysical biomarkers, and compare the advantages of each approach.
Ultimately, this research seeks to develop a personalized, integrated treatment strategy for FRDA.