Structure, gating, and regulation of the CFTR anion channel

L Csanády, P Vergani, DC Gadsby - Physiological reviews, 2019 - journals.physiology.org
The cystic fibrosis transmembrane conductance regulator (CFTR) belongs to the ATP
binding cassette (ABC) transporter superfamily but functions as an anion channel crucial for …

Small-molecule drugs for cystic fibrosis: Where are we now?

O Laselva, L Guerra, S Castellani, M Favia… - Pulmonary …, 2022 - Elsevier
The cystic fibrosis (CF) lung disease is due to the lack/dysfunction of the CF Transmembrane
Conductance Regulator (CFTR), a chloride channel expressed by epithelial cells as the …

CFTR and lung homeostasis

JF Collawn, S Matalon - American Journal of Physiology …, 2014 - journals.physiology.org
CFTR is a cAMP-activated chloride and bicarbonate channel that is critical for lung
homeostasis. Decreases in CFTR expression have dire consequences in cystic fibrosis (CF) …

[HTML][HTML] Molecular basis of cystic fibrosis: from bench to bedside

MC Dechecchi, A Tamanini… - Annals of translational …, 2018 - ncbi.nlm.nih.gov
Cystic fibrosis (CF), is an autosomal recessive disease affecting different organs. The lung
disease, characterized by recurrent and chronic bacterial infection and inflammation since …

[HTML][HTML] Correcting CFTR folding defects by small-molecule correctors to cure cystic fibrosis

M Mijnders, B Kleizen, I Braakman - Current opinion in pharmacology, 2017 - Elsevier
Highlights•Combining correctors that correct different folding defects improves
efficacy.•Complete F508del CFTR rescue likely requires full correction of NBD1.•All classes …

[HTML][HTML] Role of cystic fibrosis bronchial epithelium in neutrophil chemotaxis

G Cabrini, A Rimessi, M Borgatti, I Lampronti… - Frontiers in …, 2020 - frontiersin.org
A hallmark of cystic fibrosis (CF) chronic respiratory disease is an extensive neutrophil
infiltrate in the mucosa filling the bronchial lumen, starting early in life for CF infants. The …

Corrector VX-809 promotes interactions between cytoplasmic loop one and the first nucleotide-binding domain of CFTR

TW Loo, DM Clarke - Biochemical Pharmacology, 2017 - Elsevier
A large number of correctors have been identified that can partially repair defects in folding,
stability and trafficking of CFTR processing mutants that cause cystic fibrosis (CF). The best …

[HTML][HTML] Dysfunctional inflammation in cystic fibrosis airways: from mechanisms to novel therapeutic approaches

A Ghigo, G Prono, E Riccardi, V De Rose - International Journal of …, 2021 - mdpi.com
Cystic fibrosis (CF) is an inherited disorder caused by mutations in the gene encoding for the
cystic fibrosis transmembrane conductance regulator (CFTR) protein, an ATP-gated chloride …

[HTML][HTML] Phenotyping Rare CFTR Mutations Reveal Functional Expression Defects Restored by TRIKAFTATM

O Laselva, MC Ardelean, CE Bear - Journal of Personalized Medicine, 2021 - mdpi.com
The rare Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) mutations, c.
1826A> G (H609R) and c. 3067_3072delATAGTG (I1023_V1024del), are associated with …

Correctors of the major cystic fibrosis mutant interact through membrane-spanning domains

O Laselva, S Molinski, V Casavola, CE Bear - Molecular Pharmacology, 2018 - ASPET
The most common cystic fibrosis causing mutation is deletion of phenylalanine at position
508 (F508del), a mutation that leads to protein misassembly with defective processing …