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.
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Authors | Leonide Saad, Ilyas Washington |
Journal | Advances in experimental medicine and biology
(Adv Exp Med Biol)
Vol. 854
Pg. 355-61
( 2016)
ISSN: 0065-2598 [Print] United States |
PMID | 26427432
(Publication Type: Journal Article, Research Support, N.I.H., Extramural, Research Support, Non-U.S. Gov't)
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Chemical References |
- Phenyl Ethers
- Propanolamines
- Vitamins
- emixustat
- Vitamin A
- Deuterium
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Topics |
- Blindness
(etiology, prevention & control)
- Clinical Trials as Topic
- Deuterium
(chemistry)
- Dimerization
- Humans
- Macular Degeneration
(complications, congenital, prevention & control)
- Models, Chemical
- Molecular Conformation
(drug effects)
- Molecular Structure
- Phenyl Ethers
(therapeutic use)
- Propanolamines
(therapeutic use)
- Retinal Dystrophies
(complications, prevention & control)
- Stargardt Disease
- Vitamin A
(chemistry, therapeutic use)
- Vitamins
(chemistry, therapeutic use)
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