Article

CFTR structures bound to ETI components predict rare mutation response to modulator combinations

2024-10-16

Abstract excerpt

<h4>Background</h4> CFTR protein structures bound to each of Elexacaftor/Tezacaftor/Ivacaftor (ETI) were recently established. We aimed to use this data to predict and assess responses to ETI and each of its components in intestinal organoids derived from patients carrying rare CFTR mutations, not yet approved for ETI, based on their mutation location within the CFTR structure. <h4>Methods</h4> Organoids were gene...

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Literature Corpus work: 19cbabe5-067b-5c31-be2a-228e0fd8eae9
DOI: 10.1101/2024.10.12.24314979

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