Results 11 to 20 of about 4,681 (114)
Rapid therapeutic advances in CFTR modulator science [PDF]
Pediatric Pulmonology, 2018 AbstractCystic fibrosis (CF) is an autosomal recessive genetic disease caused by variants in the gene encoding the cystic fibrosis transmembrane conduction regulator (CFTR) protein. Loss of CFTR function disrupts chloride, bicarbonate and regulation of sodium transport, producing a cascade of mucus obstruction, inflammation, pulmonary infection, and ...
John P Clancy
openaire +4 more sources
Understanding and addressing the needs of people with cystic fibrosis in the era of CFTR modulator therapy. [PDF]
Lancet Respir Med, 2023 Hisert KB +12 more
europepmc +2 more sources
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
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
Of muscle modulation and the CFTR gate [PDF]
Journal of General Physiology, 2015 This month’s installment of Generally Physiological considers regulation of excitation–contraction coupling by PIP2 and the investigation of an appealing hypothesis for how a transporter might evolve into a channel.
openaire +2 more sources
International Journal of Molecular Sciences, 2022 Highly effective cystic fibrosis transmembrane conductance regulator (CFTR) modulators have led to dramatic improvements in lung function in many people with cystic fibrosis (PwCF). However, the efficacy of CFTR modulators may be hindered by persistent airway inflammation.
Charles Bengtson +7 more
openaire +2 more sources
State-dependent modulation of CFTR gating by pyrophosphate [PDF]
Journal of General Physiology, 2009 Cystic fibrosis transmembrane conductance regulator (CFTR) is an adenosine triphosphate (ATP)-gated chloride channel. ATP-induced dimerization of CFTR's two nucleotide-binding domains (NBDs) has been shown to reflect the channel open state, whereas hydrolysis of ATP is associated with channel closure.
Tsai, Ming-Feng +4 more
openaire +2 more sources
Frontiers in Molecular Biosciences, 2023 Most of the 2,100 CFTR gene variants reported to date are still unknown in terms of their disease liability in Cystic Fibrosis (CF) and their molecular and cellular mechanism that leads to CFTR dysfunction. Since some rare variants may respond to currently approved modulators, characterizing their defect and response to these drugs is essential for ...
Railean, Violeta +7 more
openaire +3 more sources
CFTR May Modulate AQP9 Functionality in Preeclamptic Placentas
Placenta, 2009 Preeclampsia (PE) is a hypertensive disorder unique to human pregnancy. Although its causes remain unclear, it is known that altered placental villous angiogenesis and a poorly developed fetoplacental vasculature can affect the transport functions of the syncytiotrophoblast (hST).
Castro-Parodi, Mauricio Omar +4 more
openaire +3 more sources
Clinical pharmacology of CFTR modulators
With the development of cystic fibrosis transmembrane receptor (CFTR) modulating drugs, the landscape in cystic fibrosis (CF) care has changed dramatically. These drugs enable the treatment of the underlying cause of the disease. Although CFTR modulators show an impressive clincal effect at group level in people with CF (pwCF) with specific mutations ...
openaire +2 more sources