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Article

Gene therapy for RPE65-related retinal disease

18 Oct 2018

Abstract excerpt

Significant discoveries in the etiology and pathogenesis of inherited retinal diseases (IRDs) have been made in the last few decades. Of the large number genes that cause IRDs, bi-allelic mutations in RPE65 lead to Leber Congenital Amaurosis type 2 (LCA 2), and can also result in phenotypes described as severe early childhood onset retinal dystrophy (SECORD) and Retinitis pigmentosa 20 (RP20). Following the...

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PMID: 30335549
DOI: 10.1080/13816810.2018.1533027

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