Exogenous Photoreceptor-Specific N-Glycosylated PROM1 Rescues Retinal Degeneration in Patient and Mouse Models
Ping Xu, Fuying Guo, Yuan Wang…·Xiufeng Zhong
This study demonstrates that gene therapy using AAV vectors can rescue retinal degeneration in PROM1-IRD patient and mouse models by restoring PROM1 expression and photoreceptor structure.