Results 1 to 10 of about 4,681 (114)
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
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Editorial: Real-world experience with CFTR modulator therapy. [PDF]
Front Pharmacol, 2023 Tümmler B, Burgel PR.
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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 +3 more
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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 +3 more
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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 +4 more
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CFTR modulator use in post lung transplant recipients [PDF]
The Journal of Heart and Lung Transplantation, 2021 Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) modulator therapy has previously been contraindicated in solid organ transplant recipients. This was due to lack of data and concern for interactions with immunosuppressive drug regimens. However, in post-lung transplant recipients, CFTR modulators may improve extrapulmonary manifestations of ...
Lauryn A. Benninger +2 more
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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
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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
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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
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