Results 81 to 90 of about 9,288 (216)

Innovative Therapeutic Strategies for Cystic Fibrosis: Moving Forward to CRISPR Technique

Frontiers in Pharmacology, 2018
One of the most revolutionary technologies in recent years in the field of molecular biology is CRISPR-Cas9. CRISPR technology is a promising tool for gene editing that provides researchers the opportunity to easily alter DNA sequences and modify gene ...
Michele Marangi, Giuseppa Pistritto, Giuseppa Pistritto   +2 more
doaj   +1 more source

Elexacaftor/tezacaftor/ivacaftor influences body composition in adults with cystic fibrosis: a fully automated CT-based analysis

Scientific Reports
A poor nutritional status is associated with worse pulmonary function and survival in people with cystic fibrosis (pwCF). CF transmembrane conductance regulator modulators can improve pulmonary function and body weight, but more data is needed to ...
D. Westhölter, J. Haubold, M. Welsner, L. Salhöfer, Johannes Wienker, S. Sutharsan, S. Strassburg, C. Taube, L. Umutlu, B. Schaarschmidt, S. Koitka, S. Zensen, M. Forsting, F. Nensa, R. Hosch, M. Opitz   +15 more
semanticscholar   +1 more source

Impact of extended Elexacaftor/Tezacaftor/Ivacaftor therapy on the gut microbiome in cystic fibrosis

medRxiv
Background: There is a paucity of knowledge on the longer-term effects of CF transmembrane conductance regulator (CFTR) modulator therapies upon the gut microbiome and associated outcomes.
R. Marsh, Claudio dos Santos, Alexander Yule, N. Dellschaft, Caroline L Hoad, C. Ng, G. Major, Alan R. Smyth, D. Rivett, C. van der Gast   +9 more
semanticscholar   +1 more source

Ivacaftor treatment in patients with cystic fibrosis and the G551D-CFTR mutation [PDF]

European Respiratory Review, 2013
Cystic fibrosis (CF) is an autosomal recessive lethal disease caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene that encodes for CFTR, an epithelial cell-surface expressed protein responsible for the transport of ...
Isabelle Sermet-Gaudelus
doaj  

Cystic fibrosis pathogens persist in the upper respiratory tract following initiation of elexacaftor/tezacaftor/ivacaftor therapy

Microbiology spectrum
Elexacaftor/tezacaftor/ivacaftor (ETI) therapy has revolutionized the treatment of cystic fibrosis (CF) for most affected individuals but the effects of treatment on sinus microbiota are still unknown.
Y. Hilliam, C. R. Armbruster, G. Rapsinski, C. W. Marshall, John Moore, J. Koirala, Leah Krainz, Jordan Gaston, V. Cooper, Stella E Lee, J. Bomberger   +10 more
semanticscholar   +1 more source

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

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   +1 more source

Fetal drug exposure after maternally administered CFTR modulators Elexacaftor/Tezacaftor/Ivacaftor in a rat model.

Biomedicine & pharmacotherapy = Biomedecine & pharmacotherapie
BACKGROUND The potential effects of the very effective cystic fibrosis triple combination drug, Elexacaftor/Tezacaftor/Ivacaftor (ETI) in pregnancy on prenatal development of offspring remain largely unknown.
Danni Li, Yimin Zhu, M. Donnelley, David Parsons, M. Habgood, E. Schneider-Futschik   +5 more
semanticscholar   +1 more source

Beyond the Lung. Impact of Elexacaftor/Tezacaftor/Ivacaftor on Sinonasal Disease in Children With Cystic Fibrosis

International Forum of Allergy and Rhinology
Elexacaftor/tezacaftor/ivacaftor (ETI) is a Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) therapy that improves pulmonary function and chronic rhinosinusitis (CRS) in cystic fibrosis (CF) adults with at least one copy of the F508del CFTR ...
M. Petitjean, Alexia Letierce, A. Bonnel, P. Reix, Eric Deneuville, N. Stremler, R. Luscan, V. Couloigner, Laurent Mely, K. Bessaci, G. Labbé, Christophe Marguet, Mairead Kelly-Aubert, Fatiha Hassani, I. Sermet-Gaudelus, F. Simon   +15 more
semanticscholar   +1 more source

Ivacaftor pharmacokinetics and lymphatic transport after enteral administration in rats

Frontiers in Pharmacology
Background: Ivacaftor is a modern drug used in the treatment of cystic fibrosis. It is highly lipophilic and exhibits a strong positive food effect.
Jiří Pozniak, Pavel Ryšánek, David Smrčka, Petr Kozlík, Tomáš Křížek, Jaroslava Šmardová, Anežka Nováková, Debanjan Das, Daniel Bobek, Mahak Arora, Jiří Hofmann, Tereza Doušová, Martin Šíma, Ondřej Slanař   +13 more
doaj   +1 more source

Quantification of Ivacaftor, Tezacaftor, Elexacaftor, and Lumacaftor and their active metabolites in plasma using UHPLC-MS/MS: Doors open to the application of therapeutic drug monitoring in cystic fibrosis treatment.

Journal of chromatography. B, Analytical technologies in the biomedical and life sciences
An ultra-high performance liquid chromatography-tandem mass spectrometry method was developed to quantify the cystic fibrosis transmembrane conductance regulator (CFTR) modulators ivacaftor, tezacaftor, elexacaftor, and lumacaftor and their active ...
A. Wessels, F. Elzinga, G. Koppelman, O. Akkerman, Paola Mian, D. Touw   +5 more
semanticscholar   +1 more source