Gene replacement in Retinitis Pigmentosa

2025-2027

This project is working to develop a gene replacement strategy for treating retinitis pigmentosa, a group of inherited blinding conditions affecting children and work-age adults. 

The treatment seeks to correct the underlying genetic mutations within the cells of the retina using CRISPR-based DNA editing tools, thus restoring normal gene function. The approach will be first assessed in cell and organoid models, before pre-clinical validation studies to enable clinical trials in patients.