Subretinal deposits in young patients treated with voretigene neparvovec-rzyl for RPE65-mediated retinal dystrophy. Issue 3 (14th July 2022)

• Record Type: Journal Article
• Authors: Lopez, Jennifer; Borchert, Mark; Lee, Thomas C; Nagiel, Aaron
• Is Part Of: British journal of ophthalmology. Volume 107:Issue 3(2023)
• Page Start: 299
• Page End: 301
• Publication Date: 2022-07-14
• Subjects: Retina -- Degeneration -- Treatment Surgery; Ophthalmology -- Periodicals; 617.7
• DOI: 10.1136/bjo-2022-321488
• Languages: English
• ISSNs: 0007-1161

Abstract

Abstract : 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.