Potential Therapeutic Agents Against Retinal Diseases Caused By Aberrant Metabolism Of Retinoids

INVESTIGATIVE OPHTHALMOLOGY & VISUAL SCIENCE(2016)

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Abstract
The retinoid (visual) cycle is a complex enzymatic pathway that operates in the retina for the regeneration of 11-cis-retinal (11-cis-Ral), the inherent visual chromophore indispensable for vision. Deficiencies in the retinoid metabolism are involved in pathologic mechanisms of several forms of retinal diseases including age-related macular degeneration, Stargardt's disease, and Leber's congenital amaurosis, for which no effective cures presently exist. Nevertheless, the interference of abnormal retinoid metabolism with chemicals has been considered to be a promising strategy aimed at alleviating these retinal dysfunctions. Moreover, since gene therapy is gaining increasing importance in clinical practice, the modulation of key enzymes implicated with the retinoid cycle at a genetic level will hold great promise for the treatment of patients with degenerative diseases of the retina.
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Key words
retinoid visual cycle, retinoids, metabolism, visual chromophores, photoreceptor, gene therapy, retinylamine, RPE65, retinal pigment epithelium
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