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.

中文翻译：

---

用 voretigene neparvovec-rzyl 治疗 RPE65 介导的视网膜营养不良的年轻患者的视网膜下沉积

我们报告了一系列三名年轻患者（年龄：22 个月、2 岁和 5 岁），他们在视网膜下 voretigene neparvovec-rzyl 治疗 RPE65 介导的视网膜营养不良后第一周出现视网膜下沉积。在 5 岁儿童中，双眼下缘也观察到视网膜下沉积物。所有三名患者均通过治疗改善了视觉功能，黄斑和下层视网膜下沉积物在随访期间均得到改善或消退。随着视网膜基因治疗试验数量的持续增长，这些发现可能会为基于 AAV 的基因治疗的传递参数和安全性提供信息。