Subretinal deposits in young patients treated with voretigene neparvovec-rzyl for RPE65-mediated retinal dystrophy

被引：11
作者：
Lopez, Jennifer [1 ,2 ]
Borchert, Mark [1 ,2 ]
Lee, Thomas C. [1 ,2 ]
Nagiel, Aaron [1 ,2 ]
机构：
[1] Childrens Hosp Los Angeles, Vis Ctr, Dept Surg, Los Angeles, CA 90027 USA
[2] Keck Sch Med, USC Roski Eye Inst, Dept Ophthalmol, Los Angeles, CA USA
来源：
BRITISH JOURNAL OF OPHTHALMOLOGY | 2023年 / 107卷 / 03期
关键词：
Retina; Degeneration; Treatment Surgery; GENE-THERAPY;
D O I：
10.1136/bjo-2022-321488
中图分类号：
R77 [眼科学];
学科分类号：
100212 ;
摘要：
We report a series of three young patients (ages: 22 months, 2 years, and 5 years) who developed subretinal deposits at post-operative week one following subretinal voretigene neparvovec-rzyl treatment for RPE65-mediated retinal dystrophy. In the 5-year-old, subretinal deposits were also observed in the inferior periphery of both eyes. All three patients experienced improved visual function with treatment, and both the macular and inferior subretinal deposits have improved or resolved over the follow-up period. These findings may inform the delivery parameters and safety profile of AAV-based gene therapy as the number of retinal gene therapy trials continues to grow.
引用
收藏
页码：299 / 301
页数：3
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