Article
Rescue of the Stargardt phenotype in Abca4 knockout mice through inhibition of vitamin A dimerization.
Proceedings of the National Academy of Sciences of the United States of America - 7 Jul 2015
Charbel Issa Peter, Barnard Alun R, Herrmann Philipp, Washington Ilyas, MacLaren Robert E
Abstract excerpt
Stargardt disease, an ATP-binding cassette, subfamily A, member 4 (ABCA4)-related retinopathy, is a genetic condition characterized by the accelerated accumulation of lipofuscin in the retinal pigment epithelium, degeneration of the neuroretina, and loss of vision. No approved treatment exists. Here, using a murine model of Stargardt disease, we show that the propensity of vitamin A to dimerize is responsible for...
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