Molecular Mechanisms Limiting the Therapeutic Window of AAV Gene Therapy in Mouse Models of Blue Cone Monochromacy
Brooke A. Brothers, Emily R. Sechrest, Li Ma, Madyson E. Ashcraft, Tongju Guan, Robert J. Barbera, Marion E. Cahill, Lee M. Shaw, Becky Chen, Wolfgang Baehr, Gangqing Hu, Peter Stoilov, Wen‐Tao Deng
This study evaluates AAV gene therapy in BCM mouse models, finding that older mutant mice show reduced treatment efficacy due to decreased transduction and cone degeneration, while specific promoters remain active.
bioRxiv · Feb 14, 2025 · PreprintMolecular Biology