Cockayne syndrome (CS) is characterized by growth failure and multisystemic degeneration, with a variable age of onset and rate of progression. Pathogenic variants in the ERCC6 and ERCC8 genes are known to cause this disease. Our lab works on the characterization of molecular mechanisms associated with CS, a related DNA repair disorder - xeroderma pigmentosum (XP), and a combined overlap syndrome XP-CS. We use mice, swine, and differentiated human patient iPSCs to study these diseases and test therapies. The goal of our translational program is to develop a safe and effective AAV gene therapy approach for the treatment of CS and other rare diseases, which lack established pipelines for gene therapy development.