Results 41 to 50 of about 5,299 (208)

Q1291H-CFTR molecular dynamics simulations and ex vivo theratyping in nasal epithelial models and clinical response to elexacaftor/tezacaftor/ivacaftor in a Q1291H/F508del patient

Frontiers in Molecular Biosciences, 2023
Background: Cystic fibrosis (CF) is caused by a wide spectrum of mutations in the CF transmembrane conductance regulator (CFTR) gene, with some leading to non-classical clinical presentations.
Katelin M. Allan, Katelin M. Allan, Katelin M. Allan, Miro A. Astore, Egi Kardia, Egi Kardia, Egi Kardia, Sharon L. Wong, Sharon L. Wong, Sharon L. Wong, Laura K. Fawcett, Laura K. Fawcett, Laura K. Fawcett, Laura K. Fawcett, Jessica L. Bell, Jessica L. Bell, Simone Visser, Po-Chia Chen, Renate Griffith, Adam Jaffe, Adam Jaffe, Adam Jaffe, Sheila Sivam, Orazio Vittorio, Orazio Vittorio, Serdar Kuyucak, Shafagh A. Waters, Shafagh A. Waters, Shafagh A. Waters, Shafagh A. Waters   +29 more
doaj   +1 more source

Pregnancy‐related effect on elexacaftor, tezacaftor and ivacaftor pharmacokinetics in women with cystic fibrosis

British Journal of Clinical Pharmacology, EarlyView.
Aim The number of pregnancies among women with cystic fibrosis (wwCF) has steadily increased over the past decade. However, the pharmacokinetics (PK) of elexacaftor–tezacaftor–ivacaftor (ETI) during gestation remains uncharacterized, despite its widespread use in this population.
Paulette Magnas, Naim Bouazza, Frantz Foissac, Saïk Urien, Léo Froelicher‐Bournaud, Gabrielle Lui, Victoria Guillemart, Nicolas Carlier, Jennifer Da Silva, Johanna Fesenbeckh, Reem Kanaan, Isabelle Honoré, Clémence Martin, Jean‐Marc Treluyer, Vassilis Tsatsaris, Pierre‐Régis Burgel, Sihem Benaboud   +16 more
wiley   +1 more source

A grumbling concern: A survey of gastrointestinal symptoms in cystic fibrosis in the modulator era [version 2; peer review: 1 approved, 4 approved with reservations]

NIHR Open Research
Background Gastrointestinal symptoms in cystic fibrosis (CF) are common and intrusive to daily life. Relieving gastrointestinal symptoms was identified as an important research priority and previously explored in an international survey in 2018. However,
Vinishaa Premakumar, Vigilius Gleetus, Zoe Elliott, Bu Hayee, Nicola J Rowbotham, Bethinn Evans, Helen Barr, Siobhán B Carr, Daniel Peckham, Alexander Horsley, Alan R Smyth, Natalie Goodchild, Rebecca J Calthorpe   +12 more
doaj   +1 more source

CFTR Modulator Therapy Enhances Peripheral Blood Monocyte Contributions to Immune Responses in People With Cystic Fibrosis

Frontiers in Pharmacology, 2020
BackgroundCFTR modulators decrease some etiologies of CF airway inflammation; however, data indicate that non-resolving airway infection and inflammation persist in individuals with CF and chronic bacterial infections.
Katherine B. Hisert, Katherine B. Hisert, Timothy P. Birkland, Kelly Q. Schoenfelt, Matthew E. Long, Brenda Grogan, Suzanne Carter, W. Conrad Liles, Edward F. McKone, Lev Becker, Anne M. Manicone, Sina A. Gharib   +11 more
doaj   +1 more source

Tobramycin pharmacokinetic and pharmacodynamic targets in people with cystic fibrosis

British Journal of Clinical Pharmacology, EarlyView.
Intravenous tobramycin is a first‐line treatment for Pseudomonas aeruginosa infection in people with cystic fibrosis (CF). Tobramycin exhibits concentration‐dependent activity; however, excess drug exposure can lead to nephrotoxicity and ototoxicity. While dosing typically targets serum peak (Cmax) and trough (Cmin) concentrations, the area under the ...
Kiera H. Harwood, Sarah Bannister, Stephen Duffull, April Martinez, Sarath Ranganathan, Shivanthan Shanthikumar, Amanda Gwee   +6 more
wiley   +1 more source

Peripheral Protein Quality Control as a Novel Drug Target for CFTR Stabilizer

Frontiers in Pharmacology, 2018
Conformationally defective cystic fibrosis transmembrane conductance regulator (CFTR) including rescued ΔF508-CFTR is rapidly eliminated from the plasma membrane (PM) even in the presence of a CFTR corrector and potentiator, limiting the therapeutic ...
Ryosuke Fukuda, Tsukasa Okiyoneda
doaj   +1 more source

A descriptive case series of hepatotoxicity associated with CFTR modulators and possible relevance of pharmacogenetic polymorphisms in cystic fibrosis patients

British Journal of Clinical Pharmacology, EarlyView.
Cystic fibrosis transmembrane conductance regulator (CFTR) modulators are widely used in patients with cystic fibrosis and significantly improve respiratory function and quality of life. However, their effectiveness may be limited by liver damage, which sometimes leads to treatment discontinuation, and the mechanisms underlying this remain poorly ...
Clara Laffitte Redondo, Venceslas Bourdin, Abd El Kader Ait Tayeb, Sihem Benaboud, Inès Martins, Caroline Joyau, Myriam Benhamida, Isabelle Sermet‐Gaudelus, Clémence Martin, Pierre Régis Burgel, Céline Verstuyft, Laurent Chouchana, Estelle Ayme‐Dietrich   +12 more
wiley   +1 more source

Targeting neutrophil extracellular traps in metabolic and immune niche: Nanomaterials for diabetes tissue regeneration

BMEMat, EarlyView.
The effects of NETs on regeneration of various diabetic tissues, and strategies targeting NETs for diabetes tissue regeneration. In the diabetic environment, NETs undergo complex metabolic and immune reprogramming, leading to dynamic changes in antibacterial and proinflammatory functions, and affecting regeneration of multiple systemic tissues.
Xinyi Jiang, Muxin Yue, Kai Mao, Boon Chin Heng, Noor Azlin Yahya, Yunsong Liu, Zheng Li   +6 more
wiley   +1 more source

CFTR Modulators: From Mechanism to Targeted Therapeutics

, 2022
People with cystic fibrosis (CF) suffer from a multi-organ disorder caused by loss-of-function variants in the gene encoding the epithelial anion channel cystic fibrosis transmembrane conductance regulator (CFTR). Tremendous progress has been made in both basic and clinical sciences over the past three decades since the identification of the CFTR gene.
Sheppard, David N, Yeh, Han-I, Sutcliffe, Katy, Hwang, Tzyh-Chang   +3 more
openaire   +3 more sources

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