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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Detalles Bibliográficos
Autores principales: Rong, Ziye, Gong, Xin, Hulleman, John D., Corey, David R., Mootha, V. Vinod
Formato: Online Artículo Texto
Lenguaje:English
Publicado: The Association for Research in Vision and Ophthalmology 2020
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7463221/
https://www.ncbi.nlm.nih.gov/pubmed/32934897
http://dx.doi.org/10.1167/tvst.9.9.47

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