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Adv Exp Med Biol. 2016;854:355-61. doi: 10.1007/978-3-319-17121-0_47.

Can Vitamin A be Improved to Prevent Blindness due to Age-Related Macular Degeneration, Stargardt Disease and Other Retinal Dystrophies?

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Alkeus Pharmaceuticals, Inc., 21 Drydock Ave 6th Floor, 02210, Boston, MA, USA.
Department of Ophthalmology, Columbia University Medical Center, Eye Research, 10032, New York, NY, USA.


We discuss how an imperfect visual cycle results in the formation of vitamin A dimers, thought to be involved in the pathogenesis of various retinal diseases, and summarize how slowing vitamin A dimerization has been a therapeutic target of interest to prevent blindness. To elucidate the molecular mechanism of vitamin A dimerization, an alternative form of vitamin A, one that forms dimers more slowly yet maneuvers effortlessly through the visual cycle, was developed. Such a vitamin A, reinforced with deuterium (C20-D3-vitamin A), can be used as a non-disruptive tool to understand the contribution of vitamin A dimers to vision loss. Eventually, C20-D3-vitamin A could become a disease-modifying therapy to slow or stop vision loss associated with dry age-related macular degeneration (AMD), Stargardt disease and retinal diseases marked by such vitamin A dimers. Human clinical trials of C20-D3-vitamin A (ALK-001) are underway.


A2E; ABCA4; ALK-001; AMD; Age-related macular degeneration; Bisretinoids; C20-D3-vitamin A; Lipofuscin; Retinal dystrophies; Retinaldehyde; Stargardt; Visual cycle; Vitamin A; Vitamin A dimer

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