Results 11 to 20 of about 92,053 (154)
Scientific Reports, 2017 Clinical studies with modulators of the Cystic Fibrosis Transmembrane conductance Regulator (CFTR) protein have demonstrated that functional restoration of the mutated CFTR can lead to substantial clinical benefit.
Iwona M. Pranke +17 more
doaj +2 more sources
Impact of Airway Inflammation on the Efficacy of CFTR Modulators
Cells, 2021 Defective CFTR biogenesis and activity in cystic fibrosis airways leads to airway dehydration and impaired mucociliary clearance, resulting in chronic airway infection and inflammation.
Carla M. P. Ribeiro, Martina Gentzsch
doaj +2 more sources
JCI InsightCystic fibrosis (CF) is a genetic disorder that disrupts CF transmembrane conductance regulator (CFTR) anion channels and impairs airway host defenses. Airway inflammation is ubiquitous in CF, and suppressing it has generally been considered to improve ...
Tayyab Rehman +4 more
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LifeCystic fibrosis is a multisystem disorder caused by mutations in the CFTR gene that lead to impaired ion and fluid transport across secretory epithelia.
Alessandra Ludovico, Debora Baroni
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CFTR modulators in triple combination in patients with cystic fibrosis
, 2020 Clinical efficacy of CFTR modulators in triple combination in patients with cystic fibrosis and one or two Phe508del alleles: a systematic review and meta ...
Julio Cortijo +4 more
core +11 more sources
Impact of CFTR modulators on exercise capacity in adolescents with cystic fibrosis
ERJ Open ResearchBackground Exercise capacity is an independent predictor of clinical worsening in cystic fibrosis (CF). There is limited evidence of the impact of cystic fibrosis transmembrane conductance regulator (CFTR) modulators on exercise capacity in children with
Molla Imaduddin Ahmed +4 more
doaj +2 more sources
CF fungal disease in the age of CFTR modulators [PDF]
, 2021 Fungi are increasingly recognised to have a significant role in the progression of lung disease in Cystic fibrosis with Aspergillus fumigatus the most common fungus isolated during respiratory sampling. The emergence of novel CFTR modulators has, however,
Shah, Anand +2 more
core +1 more source
Cystic Fibrosis – results of CFTR modulators in Croatia [PDF]
, 2023 Cistična fibroza najčešća je nasljedna bolest, koja skraćuje životni vijek, a uzrokuje je defekt u genu za transmembranski regulator provodljivosti cistične fibroze (eng. cystic fibrosis transmembrane regulator – CFTR).
Todorić, Ivana +36 more
core +1 more source
Restoration of CFTR function in patients with cystic fibrosis carrying the F508del-CFTR mutation [PDF]
, 2014 Restoration of BECN1/Beclin 1-dependent autophagy and depletion of SQSTM1/p62 by genetic manipulation or autophagy-stimulatory proteostasis regulators, such as cystamine, have positive effects on mouse models of human cystic fibrosis (CF). These measures
Salvadori L +112 more
core +1 more source
Mechanism-based corrector combination restores ΔF508-CFTR folding and function. [PDF]
, 2013 The most common cystic fibrosis mutation, Delta F508 in nucleotide binding domain 1 (NBD1), impairs cystic fibrosis transmembrane conductance regulator (CFTR)-coupled domain folding, plasma membrane expression, function and stability. VX-809, a promising
Okiyoneda, T. +21 more
core +1 more source