BEST1 gene therapy corrects a diffuse retina-wide microdetachment modulated by light exposure

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 detachm...

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Foilsithe in:Proc Natl Acad Sci U S A
Main Authors: 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.
Formáid: Artigo
Teanga:Inglês
Foilsithe: National Academy of Sciences 2018
Ábhair:
PNAS Plus
Rochtain Ar Líne:https://ncbi.nlm.nih.gov/pmc/articles/PMC5866594/
https://ncbi.nlm.nih.gov/pubmed/29507198
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1073/pnas.1720662115
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