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Targeting the E1 ubiquitin-activating enzyme (UBA1) improves elexacaftor/tezacaftor/ivacaftor efficacy towards F508del and rare misfolded CFTR mutants.

Cellular and molecular life sciences : CMLS - 16 Mar 2022

Borgo Christian, D'Amore Claudio, Capurro Valeria, Tomati Valeria, Sondo Elvira, Cresta Federico, Castellani Carlo, Pedemonte Nicoletta, Salvi Mauro

Abstract excerpt

The advent of Trikafta (Kaftrio in Europe) (a triple-combination therapy based on two correctors-elexacaftor/tezacaftor-and the potentiator ivacaftor) has represented a revolution for the treatment of patients with cystic fibrosis (CF) carrying the most common misfolding mutation, F508del-CFTR. This therapy has proved to be of great efficacy in people homozygous for F508del-CFTR and is also useful in individuals...

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PMID: 35292885
DOI: 10.1007/s00018-022-04215-3
PMCID: PMC8924136

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