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Towards Treatment ofStargardt Disease:Workshop Organizedand Sponsored by theFoundation FightingBlindness Avery E. SearsPaul S. BernsteinArtur V. Cideciyan2017 год

Towards Treatment of Stargardt Disease: Workshop Organized and Sponsored by the Foundation Fighting Blindness
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Аннотация: Accumulation of fluorescent metabolic byproducts of the visual (retinoid) cycle is associated with photoreceptor and retinal pigment epithelial cell death in both Stargardt disease and atrophic (nonneovascular) age-related macular degeneration (AMD). As a consequence of this observation, small molecular inhibitors of enzymes in the visual cycle were recently tested in clinical trials as a strategy to protect the retina and retinal pigment epithelium in patients with atrophic AMD. To address the clinical translational needs for therapies aimed at both diseases, a workshop organized by the Foundation Fighting Blindness was hosted by the Department of Pharmacology at Case Western Reserve University on February 17, 2017, at the Tinkham Veale University Center, Cleveland, OH, USA. Invited speakers highlighted recent advances in the understanding of the pathophysiology of Stargardt disease, in terms of its clinical characterization and the development of endpoints for clinical trials, and discussed the comparability of therapeutic strategies between atrophic age-related macular degeneration (AMD) and Stargardt disease. Investigators speculated that reducing the concentrations of visual cycle precursor substances and/or their byproducts may provide valid therapeutic options for the treatment of Stargardt disease. Here we review the workshop's presentations in the context of published literature to help shape the aims of ongoing research endeavors and aid the development of therapies for Stargardt disease.
Год издания: 2017
Авторы: Avery E. Sears, Paul S. Bernstein, Artur V. Cideciyan, Carel B. Hoyng, Peter Charbel Issa, Krzysztof Palczewski, Philip J. Rosenfeld, Srinivas R. Sadda, Ulrich Schraermeyer, Janet R. Sparrow, Ilyas Washington, Hendrik P. N. Scholl
Издательство: Association for Research in Vision and Ophthalmology
Источник: Translational Vision Science & Technology
Ключевые слова: Retinal Development and Disorders, Retinal Diseases and Treatments, Photoreceptor and optogenetics research
Другие ссылки: Translational Vision Science & Technology (PDF)
Translational Vision Science & Technology (HTML)
Europe PMC (PubMed Central) (PDF)
Europe PMC (PubMed Central) (HTML)
PubMed Central (HTML)
PubMed (HTML)