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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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| Publicado en: | Transl Vis Sci Technol |
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| Autores principales: | , , , , |
| Formato: | Artigo |
| Lenguaje: | Inglês |
| Publicado: |
The Association for Research in Vision and Ophthalmology
2020
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| Materias: | |
| Acceso en línea: | 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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