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Article

Preclinical Safety Evaluation of AAV5-hRORA Gene Therapy for Geographic Atrophy and ABCA4-Related Retinopathies

2026-08-15

Abstract excerpt

<title>Abstract</title> <p>Geographic atrophy (GA) secondary to dry age-related macular degeneration (AMD) and ABCA4-related retinopathies, including Stargardt disease (STGD1), represent leading causes of irreversible central vision loss for which no disease-modifying therapies are currently approved. Despite their distinct genetic etiologies and ages of onset, both conditions converge on shared maculopathologica...

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Literature Corpus work
581303bd-efac-5e94-86be-6c6a37b0e6b0
DOI
10.21203/rs.3.rs-10046118/v1
Open publication

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