Chorioretinal Atrophy after Voretigene Neparvovec Therapy in <italic>RPE65</italic> Mutation-Associated Inherited Retinal Disease: Longitudinal Characterization in Multimodal Imaging.

ABSTRACT:

Introduction

The detection of chorioretinal atrophy (CRA) following voretigene neparvovec (VN) therapy for RPE65-IRD highlights the need for long-term monitoring to better understand the safety and efficacy of this gene augmentation treatment. This study aimed to longitudinally assess the development of VN-associated CRA, its presentation in multimodal retinal imaging, and potential implications on visual function in patients with RPE65-IRD.

Methods

This single-center, prospective cohort study was conducted at the University of Bonn. A total of 21 patients with confirmed RPE65-IRD underwent subretinal VN therapy. Multimodal imaging, including blue-light fundus autofluorescence (BAF), near-infrared imaging (IR), near-infrared fundus autofluorescence (IRAF), and color fundus p

SUBMITTER: Sassen SH 

PROVIDER: S-EPMC12788828 | biostudies-literature | 2025 Dec

REPOSITORIES: biostudies-literature