Research Square
Preclinical Safety Evaluation of AAV5-hRORA Gene Therapy for Geographic Atrophy and ABCA4-Related Retinopathies
Preprint: experiments in animalsInterventions
Abstract
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.
The paper
Ocugen, Inc. (United States)
Research Square, 15 Aug 2026, CC BY, Preprint, not peer-reviewed
