Article

Beyond Kaftrio : mechanistic insights to maximize N1303K-CFTR rescue

2024-03-03

Abstract excerpt

<h4>Introduction</h4> N1303K is the fourth most frequent Cystic Fibrosis (CF) causing mutation. People with CF (pwCF) clinical status can be improved by Elexacaftor(ELX)/Tezacaftor(TEZ)/Ivacaftor (ETI) combotherapy. We investigated the mechanism underlying N1303K-CFTR rescue. <h4>Methods</h4> N1303K-CFTR expression and maturation was evaluated by Western Blot in cell lines and Human Nasal Epithelial Primary Cell...

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Literature Corpus work: 49126cc9-92e5-50f3-ab3a-e0b071e5bce6
DOI: 10.1101/2024.02.29.582514

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