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» OT-004 delivers RPGRIP1 gene and stabilizes visual loss in murine model
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Ocular
OT-004 delivers RPGRIP1 gene and stabilizes visual loss in murine model
May 30, 2023
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Mutations in the RPGRIP1 gene are associated with rare retinal dystrophies and most commonly with Leber congenital amaurosis (LCA) type 6, which is characterized by vision loss, among other symptoms.
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