Gene Therapy Blinding Retinal Diseases

to connect to one another after new treatments such as gene or cell therapy. KTEF funded a Career Starter Grant in my first year as Assistant Professor, allowing me to quickly gain momentum and then go on to receive significant funding from other organizations including the Baxter and Thome Foundations, the National Eye Institute, and Research to Prevent Blindness. None of these awards would have been possible without this “seed” funding from KTEF. But the generosity and support of the KTEF did not end there, with subsequent funding of our Predictive Medicine program and divisional endowment support. These mechanisms have allowed us to capitalize on our success as a retinal gene therapy center – the only one in California – and begin to devise new treatment paradigms for rare retinal diseases affecting children. The idea is that we can identify clinic patients with incurable retinal degenerations, devise personalized gene editing treatments for them, and then test these treatments on their own retinal cells in the laboratory. Looking back at how far we’ve come since setting out on this path, I truly believe the Knights have provided – and continue to provide – the critical footing I need to march ahead as we devise new approaches to curing these blinding retinal diseases.