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Trinucleotide Repeat-Targeting dCas9 as a Therapeutic Strategy for Fuchs’ Endothelial Corneal Dystrophy

PURPOSE: Fuchs’ endothelial corneal dystrophy (FECD) is the leading indication for corneal transplantation. Seventy percent of cases are caused by an intronic CTG triplet repeat expansion in the TCF4 gene that results in accumulation of pathogenic expanded CUG repeat RNA (CUG(exp)) as nuclear foci i...

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Bibliografiske detaljer
Udgivet i:Transl Vis Sci Technol
Main Authors: Rong, Ziye, Gong, Xin, Hulleman, John D., Corey, David R., Mootha, V. Vinod
Format: Artigo
Sprog:Inglês
Udgivet: The Association for Research in Vision and Ophthalmology 2020
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Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC7463221/
https://ncbi.nlm.nih.gov/pubmed/32934897
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1167/tvst.9.9.47
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