Article

Rescue of the Stargardt Disease phenotype in <i>Abca4</i> knockout mice through dietary modulation of the vitamin A receptor RBPR2

2025-07-02

Abstract excerpt

<h4>ABSTRACT</h4> Mutations in the ABCA4 gene in Stargardt disease (STGD1) causes accumulation of cytotoxic lipofuscin, resulting in RPE atrophy and photoreceptor dysfunction. One component of lipofuscin is the all- trans -retinal derivative, bisretinoid N- retinylidene- N- retinylethanolamine (A2E). Since ocular A2E biosynthesis relies on circulating all- trans -retinol bound to retinol binding protein 4 (R...

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Literature Corpus work: 523be084-8756-5152-8350-7f8d6fd95288
DOI: 10.1101/2025.06.27.662034

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