Results 191 to 200 of about 11,406 (228)
Some of the next articles are maybe not open access.
CFTR Modulator Therapy for Cystic Fibrosis
New England Journal of Medicine, 2017Cystic 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
Matrine modulates HSC70 levels and rescues ΔF508‐CFTR
Journal of Cellular Physiology, 2012AbstractCystic fibrosis transmembrane conductance regulator (CFTR) is a cAMP‐dependent Cl− channel located in the plasma membrane, and its malfunction results in cystic fibrosis (CF), the most common lethal genetic disease in Caucasians. Most CF patients carry the deletion of Phe508 (ΔF508 mutation); this mutation prevents the delivery of the CFTR to ...
BASILE, ANNA +7 more
openaire +3 more sources
Triple CFTR Modulator Therapy for Cystic Fibrosis
New England Journal of Medicine, 2018Cystic fibrosis is one of the most common life-threatening autosomal recessive disorders, affecting approximately 80,000 children and adults worldwide.1 It is caused by mutations that result in deficient or defective function of the cystic fibrosis transmembrane conductance regulator (CFTR), an anion channel that is normally present in epithelial ...
openaire +2 more sources
Impact of interruption of CFTR modulator therapies
Journal of Cystic FibrosisNovel 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 +12 more
openaire +2 more sources
CFTR Modulators for the Gastroenterologist
Journal of Pediatric Gastroenterology and Nutrition, 2023Sabina, Sabharwal, Sarah Shrager, Lusman
openaire +2 more sources
Expanding CFTR Modulator Testing to Carriers of CFTR Variants
Annals of the American Thoracic Society, 2021Preston E, Bratcher, Pamela L, Zeitlin
openaire +2 more sources
CFTR modulates lung secretory cell proliferation and differentiation
American Journal of Physiology-Lung Cellular and Molecular Physiology, 2000We have permanently reversed the lethal phenotype in the cystic fibrosis (CF) transmembrane conductance regulator (CFTR)-deficient (knockout) mouse after in utero gene therapy with an adenovirus containing the cftr gene. The gene transfer targeted somatic stem cells in the developing lung and intestine, and these epithelial surfaces demonstrated ...
J E, Larson +4 more
openaire +2 more sources
CFTR is a modulator of airway inflammation
American Journal of Physiology-Lung Cellular and Molecular Physiology, 2007although it has been 17 years since the identification of the cystic fibrosis transmembrane ion receptor (CFTR) gene and protein, it remains enigmatic how abnormalities in CFTR can cause chronic and persistent pulmonary infection and inflammation that lead to bronchiectasis and end-stage ...
openaire +2 more sources
CFTR Modulator Therapies in Cystic Fibrosis
2017Cystic fibrosis (CF), the most common life-shortening autosomal recessive disorder in Caucasians, results from mutations in the gene that encodes the cystic fibrosis transmembrane-conductance regulator (CFTR) protein. Nearly 2000 CFTR mutations have been identified as being associated with disease, with known effects of a given mutation on protein ...
David R. Spielberg +2 more
openaire +1 more source