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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?

Author information

1
Alkeus Pharmaceuticals, Inc., 21 Drydock Ave 6th Floor, 02210, Boston, MA, USA. leonide@alkeus.com.
2
Department of Ophthalmology, Columbia University Medical Center, Eye Research, 10032, New York, NY, USA. iw2101@columbia.edu.

Abstract

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.

KEYWORDS:

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

PMID:
26427432
DOI:
10.1007/978-3-319-17121-0_47
[Indexed for MEDLINE]
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