Article

A new corrector rescues F508del-CFTR folding through stabilization of the TMD1-NBD1 linker

2025-03-11

Abstract excerpt

<h4>ABSTRACT</h4> While Trikafta has turned many CF patients into people that lead relatively healthy lives, additional modulators are still needed. We developed an acylsulfonamide-type C2 corrector X307810 that is potent and shows a distinctive mode of action in rescuing F508del-CFTR from degradation at the endoplasmic reticulum. We tracked the de-novo folding and assembly of each CFTR domain with a radio-active...

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Literature Corpus work: dbe10d01-db61-5a5f-9118-7ea6b8a07bdb
DOI: 10.1101/2025.03.10.640553

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