Results 61 to 70 of about 5,785 (196)
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 +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 +4 more
openaire +1 more source
Clinical Pharmacology &Therapeutics, EarlyView.Precision medicine offers the opportunity to improve the benefit–risk profile of new therapies by prospectively identifying patients most likely to respond or least likely to experience harm; however, its systematic integration into drug development remains inconsistent outside oncology.
Ingrid Holst‐Laubjerg +1 more
wiley +1 more source
JPGN Reports, EarlyView.Abstract Objectives The efficacy of cystic fibrosis transmembrane conductance regulator (CFTR)‐modulator therapies in preventing or ameliorating cystic fibrosis liver disease (CFLD) by correcting CFTR in cholangiocytes is not well‐documented. This study aimed to assess liver function during CFTR‐modulators.
Laura Giugliano +12 more
wiley +1 more source
Neuroprotection, EarlyView.Abstract Prime editing, a novel clustered regularly interspaced short palindromic repeats (CRISPR)‐based technology, fuses a reverse transcriptase (RT) to an engineered CRISPR‐associated protein 9 (Cas9) and uses a prime editing guide RNA (pegRNA)‐encoded template.
Tianshan Ji +4 more
wiley +1 more source
Gastro Hep AdvancesBackground and Aims: Up to 40% of patients with cystic fibrosis (CF) develop CF-related liver disease (CFrLD), which can progress to the point of requiring liver transplantation (LT).
Sara Naimimohasses +6 more
doaj +1 more source
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 +4 more
wiley +1 more source
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 +20 more
wiley +1 more source
Advances in Respiratory MedicineA common life-threatening hereditary disease, Cystic Fibrosis (CF), affects primarily Caucasian infants. High sweat-salt levels are observed as a result of a single autosomal mutation in chromosome 7 that affects the critical function of the cystic ...
Saba Anwar +5 more
doaj +1 more source
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 +7 more
wiley +1 more source