Results 101 to 110 of about 5,299 (208)

Persistence and evolution of Pseudomonas aeruginosa following initiation of highly effective modulator therapy in cystic fibrosis

mBio
Today, more than 90% of people with cystic fibrosis (pwCF) are eligible for the highly effective cystic fibrosis transmembrane conductance regulator (CFTR) modulator therapy called elexacaftor/tezacaftor/ivacaftor (ETI) and its use is widespread.
Catherine R. Armbruster, Yasmin K. Hilliam, Anna C. Zemke, Samar Atteih, Christopher W. Marshall, John Moore, Junu Koirala, Leah Krainz, Jordan R. Gaston, Stella E. Lee, Vaughn S. Cooper, Jennifer M. Bomberger   +11 more
doaj   +1 more source

Development and analytical validation of a targeted short‐read next generation sequencing‐based pharmacogenetic panel for comprehensive variant detection

British Journal of Pharmacology, Volume 183, Issue 16, Page 4735-4760, August 2026.
Abstract Background and Purpose Genomic profiling of patients for genetic variants that modify the effect of specific medications has many benefits, including the possibility of avoiding toxicities and ensuring an adequate effect of the medication. Our intention was to develop a comprehensive, high‐quality pharmacogenetic test panel for clinical use ...
Anna Gréen, Nils Johan Fredriksson, Michael Andresen, Jon Jonasson, Malin Lindqvist Appell, Henrik Gréen   +5 more
wiley   +1 more source

Characterization of two ultra-rare CFTR variants, P.Leu999del and P.Glu1104Lys, with unknown theratyping profiles

Orphanet Journal of Rare Diseases
Background Individuals carrying ultra-rare CFTR variants remain untreated with CFTR modulator therapies due to a lack of functional and clinical data supporting variant-specific responsiveness. This study aimed to functionally characterize two ultra-rare
Tereza Dousova, Lucie Borek-Dohalska, Stepanka Novotna, Veronika Skalicka, Malgorzata Libik, Milan Macek, Pavel Drevinek   +6 more
doaj   +1 more source

Identification of drug repurposing candidates for the treatment of polycystic kidney disease

British Journal of Pharmacology, Volume 183, Issue 16, Page 5082-5101, August 2026.
Background and Purpose Autosomal dominant polycystic kidney disease (ADPKD) is a leading cause of end‐stage renal disease with limited treatment options. Drug repurposing offers a promising strategy to find effective treatments. Experimental Approach We identified birinapant, bardoxolone methyl and salicylic acid as repurposing candidates for ADPKD and
Alina Meyer, Paraskevi Sotiropoulou, Koussai Bouker, Dennis Svedberg, Rebekkah Hammar, Hester Bange, Per Larsson, André Mateus, Daniel R. Dietrich, Per Artursson   +9 more
wiley   +1 more source

Adenine base editing of CFTR using receptor targeted nanoparticles restores function to G542X cystic fibrosis airway epithelial cells

Cellular and Molecular Life Sciences
The cystic fibrosis (CF) causing variant G542X harbours a premature translation stop signal in the cystic fibrosis transmembrane conductance regulator (CFTR) mRNA. This results in nonsense-mediated decay and loss of functional CFTR protein which leads to
Isabelle Rose, Miriam Greenwood, Matthew Biggart, Natalie Baumlin, Robert Tarran, Stephen L. Hart, Deborah L. Baines   +6 more
doaj   +1 more source

Triple combination cystic fibrosis transmembrane receptor modulator effects on glycaemia and insulin kinetics in cystic fibrosis with and without diabetes

Internal Medicine Journal, Volume 56, Issue 8, Page 1356-1360, August 2026.
Abstract Background Greater insight into the effects of cystic fibrosis (CF) transmembrane modulators such as elexacaftor‐tezacaftor‐ivacaftor (ETI) on glucose metabolism can support a more dynamic and individualised approach to CF‐related dysglycaemia.
Yi W. Chen, Rachel Prasad, Peter G. Middleton, D. Jane Holmes‐Walker   +3 more
wiley   +1 more source

Integrated histopathological, physiological, biochemical, and molecular analysis of gill responses to acute salinity stress in red tilapia (Oreochromis spp.)

Journal of the World Aquaculture Society, Volume 57, Issue 4, August 2026.
Abstract Growing freshwater scarcity in China is driving aquaculture interests toward saline‐tolerant fish species. Red tilapia (Oreochromis spp.) is a euryhaline species with strong osmoregulatory capacity, making it an ideal model for investigating stress‐induced physiological changes.
Moustafa Saleh, Rahma Aboueleila, Mohamed Fekri Badran, Jixiang Hua, Yalun Dong, Siqi Lu, Wen‐jing Tao, Yifan Tao, Jun Qiang   +8 more
wiley   +1 more source

Editorial: Real-world experience with CFTR modulator therapy. [PDF]

Front Pharmacol, 2023
Tümmler B, Burgel PR.
europepmc   +1 more source

The gut-lung axis in the CFTR modulator era. [PDF]

Front Cell Infect Microbiol, 2023
Lussac-Sorton F, Charpentier É, Imbert S, Lefranc M, Bui S, Fayon M, Berger P, Enaud R, Delhaes L.   +8 more
europepmc   +1 more source

Increased aminotransferases do not necessarily mean hepatotoxicity of CFTR modulator therapy. [PDF]

Clin Exp Hepatol, 2023
Woynarowski M, Sapiejka E, Jóźwiak M, Wierzbicka-Rucińska A.   +3 more
europepmc   +1 more source