Gene therapies for age-related macular degeneration and uveitis  

Age-related macular degeneration (AMD) and uveitis are major causes of blindness worldwide. 

We will use the harmless virus AAV to disable a disease-causing gene in the retina that is thought to play a central role in macular degeneration. The efficacy and safety of this approach will be tested in animal models. This will also provide the opportunity for an in-depth analysis of immune responses to gene editing and optimisation of the surgical technique for delivering gene therapy to the retina in clinical trials. We will also design and test a new treatment for uveitis that uses AAV to deliver an immunomodulatory signal to the retina which could dampen down immune cell-driven chronic inflammation in the eye. 

This project will improve our understanding of the fundamental mechanisms underlying AMD and ocular ‘immune privilege’, and lead to potential new treatments for common retinal diseases.