Article

Variant-selective RNA-targeting small molecules partially restore CFTR W1282X function in Cystic Fibrosis

2026-07-03

Abstract excerpt

<title>Abstract</title> <p>RNA is an emerging therapeutic target enabling modulation of gene expression upstream of protein synthesis. Small molecules offer a powerful approach to targeting RNA by exploiting the dynamic structural landscape of the molecule. Here we show that the p.W1282X nonsense variant in CFTR, a common Class I variant causing cystic fibrosis, induces a distinct localized RNA conformation in ex...

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Literature Corpus work: e21d14ff-da8b-53a3-a8be-29544e4e0a9b
DOI: 10.21203/rs.3.rs-10093540/v1

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