Results 41 to 50 of about 1,231,622 (330)
The impact of CFTR modulators on bone health [PDF]
Pediatric Pulmonology, 2023 We are hopeful that CTFR modulation does have a positive impact of bone health, but we have concerns about this study design being used to support the hypothesis.
Elizabeth Clarke +3 more
openalex +3 more sources
Considerations for the use of inhaled antibiotics for Pseudomonas aeruginosa in people with cystic fibrosis receiving CFTR modulator therapy. [PDF]
BMJ Open Respir ResBurgel PR +10 more
europepmc +3 more sources
Personalizing CFTR modulator therapies [PDF]
, 2021 over 2000 genetic mutations that can cause CF, resulting in many phenotypes. Recently new drugs were developed that treat the disease at the origin of the problem; they enhance the function of the Cystic Fibrosis Transmembrane conductance Regulator (CFTR) protein which is not functioning properly in CF. These potentially life saving drugs are currently
openaire +2 more sources
Children, 2023 Elexacaftor/Tezacaftor/Ivacaftor (ELX/TEZ/IVA) is a new CFTR (Cystic Fibrosis Transmembrane Conductance Regulator) modulator treatment, used over the last few years, which has shown an improvement in different clinical outcomes in patients with cystic ...
Nikoletta Kapouni +3 more
doaj +1 more source
European Radiology, 2023 Objectives To investigate whether 3D phase-resolved functional lung (PREFUL)-MRI parameters are suitable to measure response to elexacaftor/tezacaftor/ivacaftor (ETI) therapy and their association with clinical outcomes in cystic fibrosis (CF) patients ...
F. Klimeš +10 more
semanticscholar +1 more source
Frontiers in Pharmacology, 2023 Background: Together with impaired mucociliary clearance, lung disease in cystic fibrosis (CF) is driven by dysregulation of innate and adaptive immunity caused by dysfunctional CFTR (Cystic Fibrosis Transmembrane Conductance Regulator), leading to ...
P. Eschenhagen +5 more
semanticscholar +1 more source
Promoter Architecture Modulates CFTR Exon 9 Skipping [PDF]
Journal of Biological Chemistry, 2003 Using hybrid minigene experiments, we have investigated the role of the promoter architecture on the regulation of two alternative spliced exons, cystic fibrosis transmembrane regulator (CFTR) exon 9 and fibronectin extra domain-A (EDB). A specific alternative splicing pattern corresponded to each analyzed promoter.
Franco, Pagani +4 more
openaire +2 more sources
Modulation of CFTR gating by permeant ions [PDF]
Journal of General Physiology, 2014 Cystic fibrosis transmembrane conductance regulator (CFTR) is unique among ion channels in that after its phosphorylation by protein kinase A (PKA), its ATP-dependent gating violates microscopic reversibility caused by the intimate involvement of ATP hydrolysis in controlling channel closure. Recent studies suggest a gating model featuring an energetic
Yeh, Han-I +2 more
openaire +2 more sources
Cureus, 2021 Cystic fibrosis (CF) is a potentially fatal genetic disease that causes serious lung damage. With time, researchers have a more complete understanding of the molecular-biological defects that underlie CF.
Anas Zaher +4 more
semanticscholar +1 more source
CFTR Modulators: From Mechanism to Targeted Therapeutics
, 2022 People with cystic fibrosis (CF) suffer from a multi-organ disorder caused by loss-of-function variants in the gene encoding the epithelial anion channel cystic fibrosis transmembrane conductance regulator (CFTR). Tremendous progress has been made in both basic and clinical sciences over the past three decades since the identification of the CFTR gene.
Sheppard, David N +3 more
openaire +4 more sources