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Gene therapy restores vison in dogs with severe form of Leber congenital amaurosis

Gene therapy restores vison in dogs with severe form of Leber congenital amaurosis Gustavo Aguirre and William Beltran, veterinary ophthalmologists and vision scientists at the University of Pennsylvania School of Veterinary Medicine, have studied a wide range of different retinal blinding disorders. But the one caused by mutations in the NPHP5 gene, leading to a form of Leber congenital amaurosis (LCA), is one of the most severe. Children with this disorder are not visual. They have a wandering, searching look on their faces and are usually diagnosed at a young age. Gustavo Aguirre, Veterinary Ophthalmologist and Vision Scientist, University of Pennsylvania School of Veterinary Medicine

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