Wu, Zhao, Wang, Zhang, Hang et al. describe a mouse model of CNGA1-retinitis pigmentosa (RP) and the effect of AAV-mediated gene augmentation. The model replicates key features of human patients, and it demonstrates that AAV-mediated gene augmentation restores vision and preserves photoreceptors.
- Yidong Wu
- Tianyuan Zhao
- Xiaodong Sun
