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Often diagnosed in childhood or adolescence, Stargardt disease is an inherited form of macular degeneration causing central vision loss

AAV vectors are generally safe at therapeutic doses eight AAV-based gene therapies have been FDA-approved as of 2024 but at high doses, above roughly 10^14 viral genomes per kilogram, the literature documents immune reactions, liver toxicity, and in rare cases, genotoxicity

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IL-2 is responsible for maintaining T-cell viability, and therefore promotes T-cell responses