Article
Oligonucleotides targeting TCF4 triplet repeat expansion inhibit RNA foci and mis-splicing in Fuchs' dystrophy.
Human molecular genetics - 15 Mar 2018
Hu Jiaxin, Rong Ziye, Gong Xin, Zhou Zhengyang, Sharma Vivek K, Xing Chao, Watts Jonathan K, Corey David R, Mootha V Vinod
Abstract excerpt
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 splicing factors and impairs splicing. We investigated the sense and antisense RNA landscape at the FECD gene and find that...
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