Results 101 to 110 of about 1,459,532 (261)

State-dependent modulation of CFTR gating by pyrophosphate [PDF]

Journal of General Physiology, 2009
Cystic fibrosis transmembrane conductance regulator (CFTR) is an adenosine triphosphate (ATP)-gated chloride channel. ATP-induced dimerization of CFTR's two nucleotide-binding domains (NBDs) has been shown to reflect the channel open state, whereas hydrolysis of ATP is associated with channel closure.
Tsai, Ming-Feng, Shimizu, Hiroyasu, Sohma, Yoshiro, Li, Min, Hwang, Tzyh-Chang   +4 more
openaire   +2 more sources

Current Cell/Organoid and Animal Models for Primary Sclerosing Cholangitis

Portal Hypertension &Cirrhosis, EarlyView.
Primary sclerosing cholangitis (PSC) is a chronic cholestatic liver disease with limited therapeutic options and a marked risk of progression to biliary fibrosis, cirrhosis, and malignancy. Progress in PSC research has been hindered by the lack of models that faithfully recapitulate the complex biliary microenvironment and disease heterogeneity ...
Qigu Yao, Sheng Cheng, Qiaoling Pan, Jiong Yu, Hongcui Cao   +4 more
wiley   +1 more source

Flavonoids in Nasal Therapeutics: Biological Activities, Delivery Challenges, and Formulation Strategies–A Systematic Review

Phytotherapy Research, EarlyView.
Overview of the therapeutic potential of flavonoids in nasal delivery systems, highlighting their benefits to the human nasal cavity, and applications in the treatment of respiratory and brain‐related diseases. ABSTRACT Flavonoids represent a significant group of secondary metabolites.
Jeniffer Viviana Ramirez Hernández, Sara Elis Bianchi, Flávia Nathiely Silveira Fachel, Elizandra Braganhol, Sheila Porto de Matos, Valquíria Linck Bassani   +5 more
wiley   +1 more source

Adenine base editing with engineered virus-like particles rescues the CFTR mutation G542X in patient-derived intestinal organoids

iScience
Summary: Cystic fibrosis (CF) is a life-shortening autosomal recessive disease, caused by loss-of-function mutations that affect the CF transmembrane conductance regulator (CFTR) anion channel.
Lucia Nicosia, Iwona Pranke, Roberta V. Latorre, Joss B. Murray, Lisa Lonetti, Kader Cavusoglu-Doran, Elise Dreano, James P. Costello, Michael Carroll, Paola Melotti, Claudio Sorio, Isabelle Sermet-Gaudelus, Martina F. Scallan, Patrick T. Harrison   +13 more
doaj   +1 more source

Comparison of clinical features of cystic fibrosis patients eligible but not on CFTR modulators to ineligible for CFTR modulators

Pediatric Pulmonology
AbstractIntroductionCystic fibrosis transmembrane conductance regulator (CFTR) modulator drugs target the underlying defect and improve CFTR function. They are a part of standard care in many countries, but not all patients are eligible for these drugs due to age and genotype. Here, we aimed to determine the characteristics of non‐eligible patients for
Halime Nayır Büyükşahin, Nagehan Emiralioğlu, Ebru Yalçın, Velat Şen, Hadice Selimoğlu Şen, Hüseyin Arslan, Azer Kılıç Başkan, Fatma Betül Çakır, Cem Fırat Koray, Aslı İmran Yılmaz, Fatih Ercan, Derya Ufuk Altıntaş, Mahir Serbes, Özlem Keskin, Elif Arık, Figen Gülen, Meral Barlık, Oğuz Karcıoğlu, Ebru Damadoğlu, Mehmet Köse, Ali Ersoy, Ayşen Bingöl, Erdem Başaran, Eylül Pınar Çakır, Ayşe Tana Aslan, Yakup Canıtez, Merve Korkmaz, Ali Özdemir, Koray Harmancı, Şule Selin Soydaş, Melih Hangül, Hasan Yüksel, Gizem Özcan, Pervin Korkmaz, Mehmet Kılıç, Zeynep Gökçe Gayretli Aydın, Gönül Çaltepe, Demet Can, Sibel Doğru, Gökçen Kartal Öztürk, Ayşe Süleyman, Erdem Topal, Beste Özsezen, Mina Hızal, Ezgi Demirdöğen, Hamza Ogun, Şermin Börekçi, Hakan Yazan, Erkan Çakır, Tuğba Şişmanlar Eyüboğlu, Nazan Çobanoğlu, Güzin Cinel, Sevgi Pekcan, Uğur Özçelik, Deniz Doğru   +54 more
openaire   +5 more sources

Update on Non‐Biological and RNA‐Based Therapeutics in Chronic Inflammatory Diseases: Precision Medicine Through Small Molecules: An EAACI Position Paper

Allergy, EarlyView.
ABSTRACT In the last decades, critical advancements in research technology and knowledge on disease mechanisms steered therapeutic approaches for chronic inflammatory diseases towards unprecedented target specificity. For allergic and chronic lung diseases, biologic drugs pioneered this goal, acquiring on the way—through the clinical use of monoclonal ...
F. Roth‐Walter, I. M. Adcock, C. Benito‐Villalvilla, R. Bianchini, L. Bjermer, G. Caramori, L. Cari, K. F. Chung, Z. Diamant, I. Eguiluz‐Gracia, E. F. Knol, M. Jesenak, F. Levi‐Schaffer, G. Nocentini, L. O'Mahony, O. Palomares, F. Redegeld, M. Sokolowska, B. Van Esch, M. Zurlo, C. Stellato   +20 more
wiley   +1 more source

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

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   +1 more source

P-84 HAVE CFTR MODULATORS CHANGED THE NEED FOR LIVER TRANSPLANTATION AMONG PATIENTS WITH CYSTIC FIBROSIS? AN ANALYSIS OF THE UNOS DATABASE

Annals of Hepatology
Conflict of interest: No Introduction and Objectives: The impact of cystic fibrosis (CF) transmembrane conductance regulator (CFTR) modulators on the natural history of liver disease is unknown.
MANUEL MENDIZABAL, Claire Cywes, Nicolas Muñoz, Amay Banker, Christian Bermudez, Denis Hadjiliadis, Peter Abt   +6 more
doaj   +1 more source

Differential Roles for IL‐4Rα and IL‐13Rα1 in Immune Cell Infiltration and Epithelial Remodeling in Experimental Eosinophilic Gastritis

Allergy, EarlyView.
This study described the development of an experimental model for eosinophilic gastritis (EoG). Experimental EoG recapitulates histopathological features of human disease. including foveolar elongation, fibrosis, eosinophilia, mastocytosis and shared transcriptional programs.
Anish Dsilva, Shraddha Sharma, Shireen Barakey, Ariel Wagner, Alice Keisar, Tali Bar‐on, Michal Itan, Ariel Munitz   +7 more
wiley   +1 more source

Immunogenic adverse events to CFTR modulators – An international survey [PDF]

Background CFTR modulator therapy has unprecedented positive effects on people with CF (pwCF). However, immunogenic reactions to CFTR modulator therapy may lead to drug discontinuation.
Behan, L, Behan, L., Stahl, M., Wisniewski, S., Naisbitt, DJ, Roehmel, J.F., Stahl, M, Wisniewski, S, Urbantat, RM, Mall, MA, Gardner, J., Naisbitt, D.J., Mall, M.A., Peckham, D, Peckham, D., Gardner, J, Roehmel, JF, Urbantat, R.M.   +17 more
core   +1 more source