Targeting Friedreich Ataxia: A Sustainable Path to Safer and Smarter Therapeutics Through Integrated Docking and Toxicology

Friedreich's ataxia (FA) is an autosomal recessive disorder affecting the nervous and cardiovascular systems, characterized by progressive ataxia, dysarthria, and muscle weakness. It results from a GAA trinucleotide repeat expansion in the FXN gene, leading to epigenetic silencing via heterochromatin formation, which reduces frataxin production-a mitochondrial protein essential for iron-sulphur cluster biogenesis and cellular energy production. Frataxin deficiency causes oxidative stress and mitochondrial dysfunction. Current treatments offer limited effectiveness, primarily addressing symptoms. Our study aims to develop novel therapeutic candidates by leveraging computational protein-ligand docking through the Galaxy EU platform, targeting frataxin deficiencies. High-throughput docking and toxicology studies have identified several promising compounds with potential therapeutic efficacy, offering hope for more effective and sustainable treatments for FA.