Article
BEST1 gene therapy corrects a diffuse retina-wide microdetachment modulated by light exposure.
Proceedings of the National Academy of Sciences of the United States of America - 20 Mar 2018
Guziewicz Karina E, Cideciyan Artur V, Beltran William A, Komáromy András M, Dufour Valerie L, Swider Malgorzata, Iwabe Simone, Sumaroka Alexander, Kendrick Brian T, Ruthel Gordon, Chiodo Vince A, Héon Elise, Hauswirth William W, Jacobson Samuel G, Aguirre Gustavo D
Abstract excerpt
Mutations in the BEST1 gene cause detachment of the retina and degeneration of photoreceptor (PR) cells due to a primary channelopathy in the neighboring retinal pigment epithelium (RPE) cells. The pathophysiology of the interaction between RPE and PR cells preceding the formation of retinal detachment remains not well-understood. Our studies of molecular pathology in the canine BEST1 disease model revealed...
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