接受 voretigene neparvovec-rzyl 治疗的 RPE65 相关视网膜营养不良的年轻患者的视网膜下沉积物。
Subretinal deposits in young patients treated with voretigene neparvovec-rzyl for RPE65-mediated retinal dystrophy.
机构信息
The Vision Center, Department of Surgery, Children's Hospital Los Angeles, Los Angeles, California, USA.
Department of Ophthalmology, USC Roski Eye Institute, Keck School of Medicine, Los Angeles, California, USA.
出版信息
Br J Ophthalmol. 2023 Mar;107(3):299-301. doi: 10.1136/bjo-2022-321488. Epub 2022 Jul 14.
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.
摘要
我们报告了三例年轻患者（年龄分别为 22 个月、2 岁和 5 岁），他们在接受雷帕霉素靶蛋白（RPE65）介导的视网膜营养不良的视网膜下注射 voretigene neparvovec-rzyl 治疗后一周出现视网膜下沉积物。在 5 岁的患者中，双眼的下周边部也观察到视网膜下沉积物。所有三名患者在治疗后视力功能均得到改善，且在随访期间，黄斑和下视网膜下沉积物均有改善或消退。这些发现可能会影响基于腺相关病毒（AAV）的基因治疗的传递参数和安全性概况，因为视网膜基因治疗试验的数量继续增加。
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