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...

Disgrifiad llawn

Wedi'i Gadw mewn:
Manylion Llyfryddiaeth
Cyhoeddwyd yn:Hum Mol Genet
Prif Awduron: Hu, Jiaxin, Rong, Ziye, Gong, Xin, Zhou, Zhengyang, Sharma, Vivek K, Xing, Chao, Watts, Jonathan K, Corey, David R, Mootha, V Vinod
Fformat: Artigo
Iaith:Inglês
Cyhoeddwyd: Oxford University Press 2018
Pynciau:
Articles
Mynediad Ar-lein: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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