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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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Bibliografski detalji
Izdano u:Transl Vis Sci Technol
Glavni autori: Rong, Ziye, Gong, Xin, Hulleman, John D., Corey, David R., Mootha, V. Vinod
Format: Artigo
Jezik:Inglês
Izdano: The Association for Research in Vision and Ophthalmology 2020
Teme:
Online pristup: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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