Gene Therapy Rescues Cone Function in an All-cone Retina Mouse Model for Blue Cone Monochromacy with the Most Common C203R Missense Mutation
Marion E. Cahill, Kathryn Chmelik, Brooke A. Brothers, Madyson E. Ashcraft, Tongju Guan, Artjola Puja, Yinxiao Xiang, Lee M. Shaw, Jianhai Du, Wen‐Tao Deng
This study shows gene therapy can restore cone function and structure in a mouse model of blue cone monochromacy with the C203R mutation.
bioRxiv · Sep 4, 2025 · PreprintMolecular Biology