Characterization of Defects in Ion Transport and Tissue Development in Cystic Fibrosis Transmembrane Conductance Regulator (CFTR)-Knockout Rats [PDF]
, 2014 Animal models for cystic fibrosis (CF) have contributed significantly to our understanding of disease pathogenesis. Here we describe development and characterization of the first cystic fibrosis rat, in which the cystic fibrosis transmembrane conductance
Bedwell, David M. +18 more
core +15 more sources
Modulator Therapy for Cystic Fibrosis: An Exploration of Current Research [PDF]
, 2020 Developing a drug therapy that addresses the root cause of cystic fibrosis (CF) by increasing CFTR protein levels has long been a research challenge. After genetic therapy failed because a suitable delivery system could not be found, researchers began ...
Rombocos, Jessalyn
core +1 more source
Advances in bronchiectasis:endotyping, genetics, microbiome, and disease heterogeneity [PDF]
, 2018 Bronchiectasis is characterised by pathological dilation of the airways. More specifically, the radiographic demonstration of airway enlargement is the common feature of a heterogeneous set of conditions and clinical presentations.
Chalmers, James D. +2 more
core +2 more sources
Robust Stimulation of W1282X-CFTR Channel Activity by a Combination of Allosteric Modulators. [PDF]
PLoS ONE, 2016 W1282X is a common nonsense mutation among cystic fibrosis patients that results in the production of a truncated Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) channel. Here we show that the channel activity of the W1282X-CFTR polypeptide is
Wei Wang +4 more
doaj +1 more source
The gut-lung axis in the CFTR modulator era
Frontiers in Cellular and Infection Microbiology, 2023 The advent of CFTR modulators represents a turning point in the history of cystic fibrosis (CF) management, changing profoundly the disease’s clinical course by improving mucosal hydration.
Florian Lussac-Sorton +22 more
doaj +1 more source
CF Fungal Disease in the Age of CFTR Modulators [PDF]
Mycopathologia, 2021 AbstractFungi 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, significantly changed the outlook of disease progression in CF.
Amelia Bercusson +2 more
openaire +4 more sources
Peroxisome Proliferator-Activated Receptor alpha (PPAR alpha) down-regulation in cystic fibrosis lymphocytes [PDF]
, 2006 Background: PPARs exhibit anti-inflammatory capacities and are potential modulators of the inflammatory response. We hypothesized that their expression and/or function may be altered in cystic fibrosis (CF), a disorder characterized by an excessive host ...
C Hubeau +53 more
core +1 more source
Functional and Pharmacological Characterization of the Rare CFTR Mutation W361R
Frontiers in Pharmacology, 2020 Understanding the functional consequence of rare cystic fibrosis (CF) mutations is mandatory for the adoption of precision therapeutic approaches for CF. Here we studied the effect of the very rare CF mutation, W361R, on CFTR processing and function.
Arnaud Billet +6 more
doaj +1 more source
The therapeutic potential of small-molecule modulators of the cystic fibrosis transmembrane conductance regulator (CFTR) Cl- channel [PDF]
, 2014 The cystic fibrosis transmembrane conductance regulator (CFTR) plays a pivotal role in fluid and electrolyte movements across ducts and tubes lined by epithelia.
Cai, Zhiwei +7 more
core +2 more sources
New drugs, new challenges in cystic fibrosis care
European Respiratory ReviewCystic fibrosis (CF) is a genetic disease caused by variants in the gene encoding for the CF transmembrane conductance regulator (CFTR) protein, a chloride and bicarbonate channel.
Isabelle Fajac +2 more
doaj +1 more source