Results 151 to 160 of about 5,816 (202)

WNK1 and WNK4 modulate CFTR activity

Biochemical and Biophysical Research Communications, 2007
The cystic fibrosis transmembrane conductance regulator (CFTR) is an ATP-gated chloride channel. WNK kinases are widely expressed modulators of ion transport. WNK1 and WNK4, two WNK kinases that are mutated in familial hyperkalemic hypertension (FHHt), are co-expressed with CFTR in several organs, raising the possibility that WNK kinases might alter ...
Chao-Ling, Yang, Xuehong, Liu, Alex, Paliege, Xiaoman, Zhu, Sebastian, Bachmann, David C, Dawson, David H, Ellison   +6 more
openaire   +2 more sources

CFTR Modulators for the Gastroenterologist

Journal of Pediatric Gastroenterology and Nutrition, 2023
Sabina, Sabharwal, Sarah Shrager, Lusman
openaire   +2 more sources

CFTR Modulator Therapies for Cystic Fibrosis

Pediatric Allergy, Immunology, and Pulmonology, 2015
The cloning of cystic fibrosis transmembrane conductance regulator (CFTR) set into motion a cascade of discoveries that have helped to reveal the underlying pathophysiologic basis of cystic fibrosis (CF). This discovery and the knowledge that followed have also provided the opportunity to target this basic defect, with the hope of reversing or ...
Aaron T, Trimble, Scott H, Donaldson
openaire   +2 more sources

Splicing Modulation as a Modifier of the CFTR Function

2006
A significant fraction of CF-causing mutations affects pre-mRNA splicing. These mutations can generate both aberrant and correct transcripts, the level of which varies among different patients. An inverse correlation was found between this level and disease severity, suggesting a role for splicing regulation as a genetic modifier.
Malka, Nissim-Rafinia, Batsheva, Kerem
openaire   +2 more sources

Expanding CFTR Modulator Testing to Carriers of CFTR Variants

Annals of the American Thoracic Society, 2021
Preston E, Bratcher, Pamela L, Zeitlin
openaire   +2 more sources

Impact of interruption of CFTR modulator therapies

Journal of Cystic Fibrosis
Novel drug therapy targeting the defective cystic fibrosis transmembrane conductance regulator protein has the potential to significantly enhance the quality of life for numerous patients with cystic fibrosis. However, in some countries social insurance does not pay for modulators because these drugs are extremely expensive.
Burcu, Capraz Yavuz, Ebru, Yalcin, Halime, Nayir Buyuksahin, Birce, Sunman, Ismail, Guzelkas, Didem, Alboga, Meltem, Akgul Erdal, Havva Ipek, Demir, Raziye, Atan, Nagehan, Emiralioglu, Deniz, Dogru, Ugur, Ozcelik, Nural, Kiper   +12 more
openaire   +2 more sources

CFTR Modulator Therapy for Cystic Fibrosis

New England Journal of Medicine, 2017
Cystic fibrosis is a disease of abnormal ion transport through epithelium that results in progressive lung disease as well as the involvement of other organs including the pancreas, gut, and liver. Cystic fibrosis is caused by mutations in the gene encoding the cystic fibrosis transmembrane conductance regulator (CFTR), and inheritance is autosomal ...
openaire   +2 more sources

Modulators of CFTR. Updates on clinical development and future directions

European Journal of Medicinal Chemistry, 2021
Anita Golec, Kate Hill, Alexandre Hinzpeter   +2 more
exaly  

CFTR Modulators: Does One Dose Fit All?

Journal of Personalized Medicine, 2021
Renske Van Der Meer, Harry G M Heijerman   +1 more
exaly  

CFTR Modulators in People with Cystic Fibrosis: Real-World Evidence in France

Cells, 2022
Espérie Burnet, Lucile RÉGARD, Pierre Regis Burgel   +2 more
exaly