From onset to blindness: a comprehensive analysis of RPGR -associated X-linked retinopathy in a large cohort in China.
Jiawen WuJunfeng LiDaowei ZhangHong-Li LiuTing LiPing XuYingke ZhaoChenchen LiFangyuan HuQian LiShenghai ZhangJi-Hong WuPublished in: Journal of medical genetics (2024)
We provide a foundation for determining the treatment window and an objective basis for evaluating the therapeutic efficacy of gene therapy for XLRP.