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

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.
AuthorsLeonide Saad, Ilyas Washington
JournalAdvances in experimental medicine and biology (Adv Exp Med Biol) Vol. 854 Pg. 355-61 ( 2016) ISSN: 0065-2598 [Print] United States
PMID26427432 (Publication Type: Journal Article, Research Support, N.I.H., Extramural, Research Support, Non-U.S. Gov't)
Chemical References
  • Phenyl Ethers
  • Propanolamines
  • Vitamins
  • emixustat
  • Vitamin A
  • Deuterium
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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