Source: The Daily Pennsylvanian
Researchers at the University of Pennsylvania’s School of Veterinary Medicine have developed advanced gene therapy mechanisms to treat inherited retinal degenerations, which cause progressive vision loss and have no current cure. Led by Raghavi Sudharsan and William Beltran, the team created four GNGT2-based gene therapy promoters designed to target the retina during late-stage degeneration. These promoters, which are active in the human eye, improve both the safety and precision of gene therapy treatments.
Building on Beltran’s earlier work in stem cell-based vision therapies, the team spent eight years developing and testing the new technology using clinically relevant models, including dogs, under ethical Veterinary care. Their findings emphasize the importance of testing therapies in realistic disease contexts rather than lab-grown tissues. Penn has filed a provisional patent on the promoters, signaling their potential for future clinical use. The researchers hope this advancement will lead to more effective treatments for both human and animal patients.
Read the full story HERE: https://www.thedp.com/article/2025/06/penn-vet-research-retinal-degeneration