Article
Stargardt disease and progress in therapeutic strategies.
Ophthalmic genetics - 1 Feb 2022
Huang Di, Heath Jeffery Rachael C, Aung-Htut May Thandar, McLenachan Samuel, Fletcher Sue, Wilton Steve D, Chen Fred K
Abstract excerpt
BACKGROUND: Stargardt disease (STGD1) is an autosomal recessive retinal dystrophy due to mutations in ABCA4, characterized by subretinal deposition of lipofuscin-like substances and bilateral centrifugal vision loss. Despite the tremendous progress made in the understanding of STGD1, there are no approved treatments to date. This review examines the challenges in the development of an effective STGD1 therapy....
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