Jinyuan Wang, Jinlu Zhang, Shicheng Yu, Hongyan Li, Shaohong Chen, Jingting Luo, Haibo Wang, Yuxia Guan, Haihan Zhang, Shiyi Yin, Huili Wang, Heping Li, Junle Liu, Jingyuan Zhu, Qiong Yang, Ying Sha, Chuan Zhang, Yuhang Yang, Xuan Yang, Xifang Zhang, Xiuli Zhao, Likun Wang, Liping Yang, Wenbin Wei
Bietti crystalline corneoretinal dystrophy is an inherited retinal disease caused by mutations in CYP4V2, which results in blindness in the working-age population, and there is currently no available treatment. Here, we report the results of the first-in-human clinical trial (NCT04722107) of gene therapy for Bietti crystalline corneoretinal dystrophy, including 12 participants who were followed up for 180-365 days. This open-label, single-arm exploratory trial aimed to assess the safety and efficacy of a recombinant adeno-associated-virus-serotype-2/8 vector encoding the human CYP4V2 protein (rAAV2/8-hCYP4V2)...
April 24, 2024: Signal Transduction and Targeted Therapy