A Phase 1/2 trial included 15 individuals in total, among them three pediatric patients. The patients all suffered from the disease Leber congenital amaurosis caused by a gene that is essential in making proteins crucial to vision.
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A Phase 1/2 trial included 15 individuals in total, among them three pediatric patients. The patients all suffered from the disease Leber congenital amaurosis caused by a gene that is essential in making proteins crucial to vision.
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