Gene therapy outcomes in young patients with RPE65-retinal degeneration.

K STEPHENSON; Abrar K. Alsalamah; Anupreet Tumber; Gareth Mercer; Brian G. Ballios; Rajeev H Muni; Erika Tavares; Peter John Kertes; Ajoy Vincent; Elise Héon

doi:10.1016/j.jcjo.2026.01.016

February 9, 2026

Gene therapy outcomes in young patients with RPE65-retinal degeneration.

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Key Points

The study aims to evaluate the effectiveness and safety of gene therapy in young patients with RPE65-related retinal degeneration.
Assess retinal sensitivity in patients treated with VN gene therapy
Monitor for post-treatment complications, including CRA
Focus on patients aged under 21 years
Gene therapy significantly improves retinal sensitivity in the treated cohort
Occurrence of CRA is noted but does not compromise the treatment's overall effectiveness
Treatment is considered safe for patients under 21

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Abstract

VN treatment is safe and effective in improving retinal sensitivity in patients under 21 years old with RPE65-LCA despite the occurrence of CRA post-treatment.

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Cite This Study

STEPHENSON et al. (2026) studied this question.

synapsesocial.com/papers/6992b3fb9b75e639e9b08dac https://doi.org/10.1016/j.jcjo.2026.01.016

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Gene therapy outcomes in young patients with RPE65-retinal degeneration.

Key Points

Ask about this paper

Abstract

Discussion

Cite This Study

Gene therapy outcomes in young patients with RPE65-retinal degeneration.

Key Points

Ask about this paper

Abstract

Discussion

Cite This Study

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