Gene Therapy for Cockayne Syndrome

Cockayne syndrome (CS) is a rare genetic disorder caused by problems in DNA repair. It leads to accelerated aging and severe neurodegenerative issues, such as cognitive impairment, nerve damage, brain atrophy, and dementia. This project aims to develop a gene therapy for the most common type of CS, known as CS type B (CSB). The research team plans to use mice to explore a virus-based delivery system for the therapy. If successful, this approach will be tested in cells, mice, and a large animal model, with the ultimate goal of applying it to human patients.