Oral levodopa rescues retinal morphology and visual function in a murine model of human albinism. (2nd April 2019)

• Record Type: Journal Article
• Authors: Lee, Helena; Scott, Jennifer; Griffiths, Helen; Self, Jay E.; Lotery, Andrew
• Is Part Of: Pigment cell & melanoma research. Volume 32:Number 5(2019)
• Page Start: 657
• Page End: 671
• Publication Date: 2019-04-02
• Subjects: albinism -- levodopa -- neuronal plasticity -- ocular -- retina -- therapeutics -- vision; Melanoma -- Periodicals; Chromatophores -- Periodicals; Animal pigments -- Periodicals; 616.99477
• DOI: 10.1111/pcmr.12782
• Languages: English
• ISSNs: 1755-1471

Abstract

Abstract: Albinism is a group of disorders characterized by pigment deficiency and abnormal retinal development. Despite being a common cause for visual impairment worldwide, there is a paucity of treatments and patients typically suffer lifelong visual disability. Residual plasticity of the developing retina in young children with albinism has been demonstrated, suggesting a post‐natal window for therapeutic rescue. L‐3, 4 dihydroxyphenylalanine (L‐DOPA), a key signalling molecule which is essential for normal retinal development, is known to be deficient in albinism. In this study, we demonstrate for the first time that post‐natal L‐DOPA supplementation can rescue retinal development, morphology and visual function in a murine model of human albinism, but only if administered from birth or 15 days post‐natal age.