Filters: Department: "Ophthalmology" Format: "application/pdf" Collection: "ir_uspace" Subject: "Genetic Vectors" Type: "Text"
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Baehr, Wolfgang | Pharmacological and rAAV gene therapy rescue of visual functions in a blind mouse model of Leber congenital amaurosis | BACKGROUND: Leber congenital amaurosis (LCA), a heterogeneous early-onset retinal dystrophy, accounts for approximately 15% of inherited congenital blindness. One cause of LCA is loss of the enzyme lecithin:retinol acyl transferase (LRAT), which is required for regeneration of the visual photopigmen... | Disease Models, Animal; Genetic Vectors; Molecular Sequence Data | 2005 |
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