Novel CFTR modulator combinations maximise rescue of G85E and N1303K in rectal organoids
ERJ Open Research, 2022 Introduction Cystic fibrosis (CF) is a severe monogenic disorder caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.
Marjolein M. Ensinck +9 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
Chloride channels regulate differentiation and barrier functions of the mammalian airway. [PDF]
, 2020 The conducting airway forms a protective mucosal barrier and is the primary target of airway disorders. The molecular events required for the formation and function of the airway mucosal barrier, as well as the mechanisms by which barrier dysfunction ...
Caplan, Michael J +14 more
core +2 more sources
Neuromodulation: Technology at the Neural Interface, EarlyView., 2021 Abstract Introduction Deep brain stimulation (DBS) is an effective treatment for refractory obsessive–compulsive disorder (OCD). Neuropsychological assessment contributes to DBS treatment in several ways: it monitors the cognitive safety of the treatment, identifies beneficial or detrimental cognitive side effects and it could aid to explain ...
Tim A. M. Bouwens van der Vlis +7 more
wiley +1 more source
Proceedings: Regenerative Medicine for Lung Diseases: A CIRM Workshop Report. [PDF]
, 2017 The mission of the California Institute of Regenerative Medicine (CIRM) is to accelerate treatments to patients with unmet medical needs. In September 2016, CIRM sponsored a workshop held at the University of California, Los Angeles, to discuss ...
DeWitt, Natalie D +2 more
core +1 more source
CFTR Modulates Orai1 Dynamics [PDF]
Biophysical Journal, 2011 Calcium signaling regulates many cellular processes and is upregulated in cystic fibrosis (CF) epithelial cells. Agonists stimulate Ca2+ release from ER stores and trigger the formation of a complex between the plasma membrane (PM) Ca2+ channel Orai1 and stromal interaction molecule 1 (STIM1), a primarily endoplasmic reticulum (ER) resident protein ...
AbuArish, Asmahan +4 more
openaire +1 more source
Attenuation of Phosphorylation-dependent activation of Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) by disease-causing mutations at the transmission interface [PDF]
, 2016 Cystic fibrosis transmembrane conductance regulator (CFTR) is a multidomain membrane protein that functions as a phosphorylation-regulated anion channel. The interface between its two cytosolic nucleotide binding domains and coupling helices conferred by
Bear, C.E. +6 more
core +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 +3 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
Some gating potentiators, including VX-770, diminish ΔF508-CFTR functional expression. [PDF]
, 2014 Cystic fibrosis (CF) is caused by mutations in the CF transmembrane regulator (CFTR) that result in reduced anion conductance at the apical membrane of secretory epithelia.
Apaja, Pirjo M. +12 more
core +1 more source