Molecular therapy : the journal of the American Society of Gene Therapy
Gene therapy for Bietti crystalline corneoretinal dystrophy: A phase 1/2 clinical trial.
Liping Yang, Dongjun Xing, Jinlu Zhang, Boshi Liu, Xiaoshuang Jiang, Shaohong Chen, Wang Li, Baoyuan Qu, Jing Qiao, Xuefeng Feng, Haicun Jia, Xianjun Hu, Shicheng Yu, Ruixuan Jia, Likun Wang
Published: 202610.1016/j.ymthe.2026.01.005
Abstract
Bietti crystalline corneoretinal dystrophy (BCD) is an inherited retinal degeneration caused by bi-allelic variants in the CYP4V2 gene. Here, we report a phase 1/2, dose-escalation clinical study to assess the safety and efficacy of ZVS101e, an adeno…
Preview only. Read the full abstract at the source