Oligonucleotides targeting TCF4 triplet repeat expansion inhibit RNA foci and mis-splicing in Fuchs’ dystrophy

Fuchs’ endothelial corneal dystrophy (FECD) is the most common repeat expansion disorder. FECD impacts 4% of U.S. population and is the leading indication for corneal transplantation. Most cases are caused by an expanded intronic CUG tract in the TCF4 gene that forms nuclear foci, sequesters splicin...

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Publié dans:Hum Mol Genet
Auteurs principaux: Hu, Jiaxin, Rong, Ziye, Gong, Xin, Zhou, Zhengyang, Sharma, Vivek K, Xing, Chao, Watts, Jonathan K, Corey, David R, Mootha, V Vinod
Format: Artigo
Langue:Inglês
Publié: Oxford University Press 2018
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Articles
Accès en ligne:https://ncbi.nlm.nih.gov/pmc/articles/PMC5886168/
https://ncbi.nlm.nih.gov/pubmed/29325021
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1093/hmg/ddy018
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