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Article

Structure-guided combination of novel CFTR correctors to improve the function of F508del-CFTR in airway epithelial cells.

Biochemical pharmacology - 1 Oct 2025

Lunaccio Dario, Allegretta Caterina, Pesce Emanuela, Scarano Naomi, Vinci Virginia, Salis Annalisa, Tasso Bruno, Brullo Chiara, Capitanio Nazzareno, Piccoli Claudia, Pedemonte Nicoletta, Cichero Elena, Millo Enrico, Laselva Onofrio

Abstract excerpt

Although remarkable rescue has been achieved for treatment of Cystic Fibrosis (CF) by the combination of two correctors (VX-661, VX-445) and one potentiator (VX-770), the stability and trafficking defects induced by the most common mutation, F508del, are not completely reversed. Therefore, more effective CFTR correctors are still needed. We employed in silico and molecular modelling approaches to design and probe...

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Source

Identifiers

PMID: 40645602
DOI: 10.1016/j.bcp.2025.117127

Genes

CFTR

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