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Duplex RNAs and ss-siRNAs Block RNA Foci Associated with Fuchs' Endothelial Corneal Dystrophy

24 Jan 2019

Abstract excerpt

Fuchs' endothelial corneal dystrophy (FECD) leads to vision loss and is one of the most common inherited eye diseases. Corneal transplants are the only curative treatment available, and there is a major unmet need for treatments that are less invasive and independent of donor tissue. Most cases of FECD are associated with an expanded CUG repeat within the intronic region of TCF4 and the mutant RNA has been...

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PMID: 30676271
DOI: 10.1089/nat.2018.0764

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Duplex RNAs and ss-siRNAs Block RNA Foci Associated with Fuchs' Endothelial Corneal DystrophyPMID 30676271Read ↗

Oligonucleotides targeting TCF4 triplet repeat expansion inhibit RNA foci and mis-splicing in Fuchs' dystrophy.Semantic proximity Read ↗

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Analyzing Presymptomatic Tissue to Gain Insights into the Molecular and Mechanistic Origins of Late-Onset Degenerative Trinucleotide Repeat DiseaseSemantic proximity Read ↗

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A common trinucleotide repeat expansion within the transcription factor 4 (TCF4, E2-2) gene predicts Fuchs corneal dystrophy.Semantic proximity Read ↗

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