Results 31 to 40 of about 1,208,918 (256)

Robust Stimulation of W1282X-CFTR Channel Activity by a Combination of Allosteric Modulators. [PDF]

PLoS ONE, 2016
W1282X is a common nonsense mutation among cystic fibrosis patients that results in the production of a truncated Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) channel. Here we show that the channel activity of the W1282X-CFTR polypeptide is
Wei Wang, Jeong S Hong, Andras Rab, Eric J Sorscher, Kevin L Kirk   +4 more
doaj   +3 more sources

Mitochondrial Fragmentation Induced by the CFTR Modulators Lumacaftor and Ivacaftor in Immortalized Cystic Fibrosis Cell Lines [PDF]

Cells
Cystic fibrosis (CF) is an autosomal recessive disease caused by mutations in the CFTR gene, which encodes a cAMP-activated chloride channel essential for epithelial function.
Camila Dib, Pablo A. Iglesias González, María de los Ángeles Aguilar, Guillermo L. Taminelli, Tatiana Limpias del Valle, Nadia E. Nuñez, Analía G. Karadayian, Tomás A. Santa-Coloma, Ángel G. Valdivieso   +8 more
doaj   +2 more sources

Epithelial responses to CFTR modulators are improved by inflammatory cytokines and impaired by antiinflammatory drugs [PDF]

JCI Insight
Cystic fibrosis (CF) is a genetic disorder that disrupts CF transmembrane conductance regulator (CFTR) anion channels and impairs airway host defenses. Airway inflammation is ubiquitous in CF, and suppressing it has generally been considered to improve ...
Tayyab Rehman, Alejandro A. Pezzulo, Andrew L. Thurman, Rachel L. Zemans, Michael J. Welsh   +4 more
doaj   +2 more sources

Effect of CFTR modulators on glucose homeostasis in children and young adults with cystic fibrosis-related diabetes: a systematic review [PDF]

Frontiers in Endocrinology
IntroductionCystic fibrosis (CF) is an autosomal recessive disorder caused by mutations in the CFTR gene, leading to impaired chloride transport, thickened mucus, and multiorgan dysfunction. Among its complications, cystic fibrosis-related diabetes (CFRD)
Paola Giordano, Giuseppina Leonetti, Vanja Granberg, Rosa Maria Pia Casolino, Giuseppe Lassandro, Maurizio Delvecchio, Giovanna Linguiti   +6 more
doaj   +2 more sources

Treatment of cystic fibrosis airway cells with CFTR modulators reverses aberrant mucus propertiesviahydration [PDF]

European Respiratory Journal, 2021
Question Cystic fibrosis (CF) is characterised by the accumulation of viscous adherent mucus in the lungs. While several hypotheses invoke a direct relationship with cystic fibrosis transmembrane conductance regulator (CFTR) dysfunction (i.e.
Cameron B. Morrison, Kendall M. Shaffer, Kenza C. Araba, Matthew R. Markovetz, Jason A. Wykoff, Nancy L. Quinney, Shuyu Hao, M. Delion, Alexis Flen, Lisa C. Morton, Jimmy C. Liao, David B. Hill, Mitchell L. Drumm, Wanda K. O’Neal, Mehmet Kesımer, Martina Gentzsch, Camille Ehré   +16 more
openalex   +2 more sources

CFTR modulators response of S737F and T465N CFTR variants on patient-derived rectal organoids [PDF]

Orphanet Journal of Rare Diseases
Background Predictions based on patient-derived materials of CFTR modulators efficacy have been performed lately in patient-derived cells, extending FDA-approved drugs for CF patients harboring rare variants.
Karina Kleinfelder, Paola Melotti, Anca Manuela Hristodor, Cristina Fevola, Giovanni Taccetti, Vito Terlizzi, Claudio Sorio   +6 more
doaj   +2 more sources

Current state of CFTR modulators for treatment of Cystic Fibrosis [PDF]

Current opinion in pharmacology (Print), 2022
Katherine A. Despotes, Scott H. Donaldson   +1 more
openalex   +2 more sources

Effect of CFTR Modulators on Oxidative Stress and Autophagy in Non-CFTR-Expressing Cells. [PDF]

Int J Mol Sci
Scialò F, Cernera G, Polise L, Castaldo G, Amato F, Villella VR.   +5 more
europepmc   +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

CFTR Modulators Therapy Efficacy in Reducing Cystic Fibrosis (CF) Exacerbation and Improving Selected Spirometry Parameters: A Real-Life Study in a Single-Centre Polish Population. [PDF]

J Clin Med
Winiarska HM, Springer D, Wojtaś F, Wysocka E, Cofta S.   +4 more
europepmc   +3 more sources