[HTML][HTML] Allosteric folding correction of F508del and rare CFTR mutants by elexacaftor-tezacaftor-ivacaftor (Trikafta) combination
G Veit, A Roldan, MA Hancock, DF Da Fonte, H Xu… - JCI insight, 2020 - ncbi.nlm.nih.gov
Based on its clinical benefits, Trikafta—the combination of folding correctors VX-661
(tezacaftor), VX-445 (elexacaftor), and the gating potentiator VX-770 (ivacaftor)—was FDA …
(tezacaftor), VX-445 (elexacaftor), and the gating potentiator VX-770 (ivacaftor)—was FDA …
SARS-CoV-2 (COVID-19) and cystic fibrosis
BA Stanton, TH Hampton… - American Journal of …, 2020 - journals.physiology.org
Cystic fibrosis (CF) is a genetic disease caused by mutations in the CFTR gene. Although
viral respiratory tract infections are, in general, more severe in patients with CF compared …
viral respiratory tract infections are, in general, more severe in patients with CF compared …
The role of CFTR in the eye, and the effect of early highly effective modulator treatment for cystic fibrosis on eye health
EK Schneider-Futschik, Y Zhu, D Li… - Progress in Retinal and …, 2024 - Elsevier
Cystic fibrosis transmembrane conductance regulator (CFTR) is a protein that plays a crucial
role in various human organs, including the respiratory and digestive systems. Dysfunctional …
role in various human organs, including the respiratory and digestive systems. Dysfunctional …
Proteostasis regulators in cystic fibrosis: current development and future perspectives
I Brusa, E Sondo, F Falchi, N Pedemonte… - Journal of Medicinal …, 2022 - ACS Publications
In cystic fibrosis (CF), the deletion of phenylalanine 508 (F508del) in the CF transmembrane
conductance regulator (CFTR) leads to misfolding and premature degradation of the mutant …
conductance regulator (CFTR) leads to misfolding and premature degradation of the mutant …
L1077P CFTR pathogenic variant function rescue by Elexacaftor–Tezacaftor–Ivacaftor in cystic fibrosis patient-derived air–liquid interface (ALI) cultures and organoids …
S Lo Cicero, G Castelli, G Blaconà, SM Bruno… - Respiratory …, 2023 - Springer
Cystic fibrosis (CF) is caused by defects of the cystic fibrosis transmembrane conductance
regulator (CFTR) gene. CFTR-modulating drugs may overcome specific defects, such as the …
regulator (CFTR) gene. CFTR-modulating drugs may overcome specific defects, such as the …
[HTML][HTML] L1077P CFTR pathogenic variant function rescue by Elexacaftor–Tezacaftor–Ivacaftor in cystic fibrosis patient-derived air–liquid interface (ALI) cultures and …
SL Cicero, G Castelli, G Blaconà, SM Bruno… - Respiratory …, 2023 - ncbi.nlm.nih.gov
Cystic fibrosis (CF) is caused by defects of the cystic fibrosis transmembrane conductance
regulator (CFTR) gene. CFTR-modulating drugs may overcome specific defects, such as the …
regulator (CFTR) gene. CFTR-modulating drugs may overcome specific defects, such as the …
Revisiting CFTR interactions: old partners and new players
CM Farinha, M Gentzsch - International Journal of Molecular Sciences, 2021 - mdpi.com
Remarkable progress in CFTR research has led to the therapeutic development of
modulators that rescue the basic defect in cystic fibrosis. There is continuous interest in …
modulators that rescue the basic defect in cystic fibrosis. There is continuous interest in …
The effect of sodium bicarbonate, a beneficial adjuvant molecule in cystic fibrosis, on bronchial epithelial cells expressing a wild-type or mutant CFTR channel
Clinical and experimental results with inhaled sodium bicarbonate as an adjuvant therapy in
cystic fibrosis (CF) are promising due to its mucolytic and bacteriostatic properties, but its …
cystic fibrosis (CF) are promising due to its mucolytic and bacteriostatic properties, but its …
A proteomic survey of the cystic fibrosis transmembrane conductance regulator surfaceome
The aim of this review article is to collate recent contributions of proteomic studies to cystic
fibrosis transmembrane conductance regulator (CFTR) biology. We summarize advances …
fibrosis transmembrane conductance regulator (CFTR) biology. We summarize advances …
How to determine the mechanism of action of CFTR modulator compounds: A gateway to theranostics
MD Amaral - European Journal of Medicinal Chemistry, 2021 - Elsevier
The greatest challenge of 21st century biology is to fully understand mechanisms of disease
to drive new approaches and medical innovation. Parallel to this is the huge biomedical …
to drive new approaches and medical innovation. Parallel to this is the huge biomedical …