Retinal Gene Therapy Trial Underway at McGill University

Our dedicated researchers worked hard over the holidays, making great progress on the PBD-ZSD retinal gene therapy project at McGill University. The PEX1 mice have received retinal injections of the normal PEX1 gene. The mice are doing well and soon we will be able to determine if this treatment has an effect on their vision.

This project is taking place in Dr. Braverman’s laboratory in collaboration with Dr. Jean Bennet (University of Pennsylvania), who is an international pioneer in retinal gene therapy. Dr. Bennet has designed the gene therapy tools, and the research team has delivered the normal PEX1 gene to the retina of our PEX1-G843D mouse.

Cheers to the amazing skills of Dr. Braverman, Dr. Jenny Song and the whole research team! We are so grateful for your passion for helping our children.