Full-field Scotopic Threshold Improvement after Voretigene Neparvovec-rzyl Treatment Correlates with Chorioretinal Atrophy

Article Ophthalmology

Development of retinal atrophy after subretinal gene therapy with voretigene neparvovec

Felix Friedrich Reichel et al.

Summary: This study evaluated the development of retinal atrophy in patients treated with Voretigene neparvovec (VN) gene therapy. It was found that retinal atrophy occurred in all eyes, primarily within the detached area. Fundus autofluorescence was identified as an important tool for monitoring atrophic changes in these patients. Interestingly, the functional outcomes of the treatment remained stable despite the presence of atrophy in central areas.

BRITISH JOURNAL OF OPHTHALMOLOGY (2023)

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Review Ophthalmology

Therapy with voretigene neparvovec. How to measure success?

Krunoslav Stingl et al.

Summary: Retinal gene supplementation therapy restores visual functions by delivering a functioning copy of the missing gene. Traditional diagnostic and functional tests cannot be applied to define subtle changes in visual functions, requiring new retinal diagnostic methods. This manuscript presents a combination of psychophysics, objective measures, and retinal imaging as an evaluation workflow for the success of gene therapy in patients with photoreceptor diseases.

PROGRESS IN RETINAL AND EYE RESEARCH (2023)

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Article Ophthalmology

Spatial and temporal resolution of the photoreceptors rescue dynamics after treatment with voretigene neparvovec

Krunoslav Stingl et al.

Summary: In this study, Voretigene Neparvovec was used to treat seven eyes of five patients with bi-allelic RPE65 mutations, showing improvements in rod function in most patients at 1 month. Visual acuity either slightly improved or remained stable, with cone function improvement in three eyes. Correlations were found between age and certain functional changes, suggesting potential parameters for therapy success.

BRITISH JOURNAL OF OPHTHALMOLOGY (2022)

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Article Ophthalmology

Inflammation after Voretigene Neparvovec Administration in Patients with RPE65-Related Retinal Dystrophy

Line Kessel et al.

Summary: This study retrospectively analyzed the prevalence of intraocular inflammation in patients receiving subretinal gene therapy. The results showed that vitritis was observed in 9 out of 23 eyes, and some eyes also had outer retinal infiltrates. The inflammation could be alleviated with immunosuppressant therapy, but close monitoring is necessary. Inflammation did not have a significant adverse effect on visual function, but it could lead to atrophy of the retinal pigment epithelium and outer retina.

OPHTHALMOLOGY (2022)

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Article Ophthalmology

Perifoveal Chorioretinal Atrophy after Subretinal Voretigene Neparvovec-rzyl for RPE65-Mediated Leber Congenital Amaurosis

William S. Gange et al.

Summary: This study retrospectively analyzed the development of perifoveal chorioretinal atrophy in patients who received VN injection. The study found that patients developed progressive atrophy after surgery, which enlarged over time. This complication may be related to ocular, surgical, and vector-related factors.

OPHTHALMOLOGY RETINA (2022)

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Article Multidisciplinary Sciences