Results 21 to 30 of about 12,522 (293)

Review of CFTR modulators 2020 [PDF]

Pediatric Pulmonology, 2021
AbstractCystic fibrosis transmembrane conductance regulator (CFTR) modulators are small molecules that directly impact the CFTR protein, improving the function of the CFTR chloride and bicarbonate channel. Beginning in 2012 with the Food and Drug Administration approval of the first CFTR modulator, ivacaftor, this class of medications has had largely ...
Danielle M. Goetz, Adrienne P. Savant
openaire   +2 more sources

Therapeutic Approaches for Patients with Cystic Fibrosis Not Eligible for Current CFTR Modulators

Cells, 2021
Cystic fibrosis is a severe autosomal recessive disorder caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene encoding the CFTR protein, a chloride channel expressed in many epithelial cells.
Isabelle Fajac, Isabelle Sermet
doaj   +1 more source

CFTR Modulator Therapy for Rare CFTR Mutants

Journal of Respiration, 2022
Cystic fibrosis (CF), the most common genetic disease among the Caucasian population, is caused by mutations in the gene encoding for the CF transmembrane conductance regulator (CFTR), a chloride epithelial channel whose dysfunction results in severe airway obstruction and inflammation, eventually leading to respiratory failure.
Marco Mergiotti, Alessandra Murabito, Giulia Prono, Alessandra Ghigo   +3 more
openaire   +2 more sources

A Novel Co-Culture Model Reveals Enhanced CFTR Rescue in Primary Cystic Fibrosis Airway Epithelial Cultures with Persistent Pseudomonas aeruginosa Infection

Cells, 2023
People with cystic fibrosis (pwCF) suffer from chronic and recurring bacterial lung infections that begin very early in life and contribute to progressive lung failure.
Deborah M. Cholon, Matthew A. Greenwald, Matthew G. Higgs, Nancy L. Quinney, Susan E. Boyles, Suzanne L. Meinig, John T. Minges, Ashlesha Chaubal, Robert Tarran, Carla M. P. Ribeiro, Matthew C. Wolfgang, Martina Gentzsch   +11 more
doaj   +1 more source

Pharmacological Responses of the G542X-CFTR to CFTR Modulators

Frontiers in Molecular Biosciences, 2022
Cystic fibrosis (CF) is a lethal hereditary disease caused by loss-of-function mutations of the chloride channel cystic fibrosis transmembrane conductance regulator (CFTR). With the development of small-molecule CFTR modulators, including correctors that facilitate protein folding and expression and potentiators that promote channel activity, about 90%
Xinxiu Fang, Jiunn-Tyng Yeh, Tzyh-Chang Hwang, Tzyh-Chang Hwang   +3 more
openaire   +3 more sources

Pharmacokinetic variability of CFTR modulators from standard and alternative regimens. [PDF]

Pulm Pharmacol Ther
Rose NR, Chalamalla AR, Garcia BA, Krick S, Bergeron J, Sadeghi H, Schellhase DE, Ryan KJ, Dowell AE, Acosta EP, Guimbellot JS.   +10 more
europepmc   +3 more sources

Enhanced Expression of Human Epididymis Protein 4 (HE4) Reflecting Pro-Inflammatory Status Is Regulated by CFTR in Cystic Fibrosis Bronchial Epithelial Cells

Frontiers in Pharmacology, 2021
Decreased human epididymis protein 4 (HE4) plasma levels were reported in cystic fibrosis (CF) patients under CFTR potentiator ivacaftor therapy, which inversely correlated with lung function improvement.
Zsolt Bene, Zsolt Bene, Zsolt Bene, Zsolt Fejes, Zsolt Fejes, Tibor Gabor Szanto, Ferenc Fenyvesi, Judit Váradi, Luka A. Clarke, Gyorgy Panyi, Milan Macek, Margarida D. Amaral, István Balogh, István Balogh, Béla Nagy, Béla Nagy   +15 more
doaj   +1 more source

CFTR modulators: transformative therapies for cystic fibrosis [PDF]

Journal of Managed Care & Specialty Pharmacy, 2021
DISCLOSURES: No funding contributed to the writing of this commentary. Both authors are employed by the Cystic Fibrosis Foundation. The Cystic Fibrosis Foundation has entered into therapeutic development award agreements and licensing agreements to assist with the development of CFTR modulators that may result in intellectual property rights, royalties,
Mary, Dwight, Bruce, Marshall
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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, Balghi, Haouaria, Arane, Tal, Wiseman, Paul, Hanrahan, John   +4 more
openaire   +1 more source

Lived experiences of individuals with cystic fibrosis on CFTR-modulators

BMC Pulmonary Medicine, 2022
Background CFTR-modulators are a category of drugs that facilitate trafficking and opening of the abnormal CFTR protein in individuals with cystic fibrosis (CF) who have certain genetic mutations.
Annelise Page, Aaron Goldenberg, Anne L. Matthews   +2 more
doaj   +1 more source