Results 21 to 30 of about 5,299 (208)
Chronic rhinosinusitis in patients with cystic fibrosis—Current management and new treatments
Laryngoscope Investigative Otolaryngology, 2020 Objectives The purpose of this article is to provide a state‐of‐the‐art review of the literature and summarize the latest publications on medical and surgical treatment of cystic fibrosis (CF) chronic rhinosinusitis (CRS), with an emphasis on describing ...
Brian Jake Johnson +2 more
doaj +1 more source
The Journal of Clinical Investigation, 2022 The vast majority of people with cystic fibrosis (CF) are now eligible for CF transmembrane regulator (CFTR) modulator therapy. The remaining individuals with CF harbor premature termination codons (PTCs) or rare CFTR variants with limited treatment ...
Rhianna E. Lee +15 more
doaj +1 more source
Modulation of CFTR gating by permeant ions [PDF]
Journal of General Physiology, 2014 Cystic fibrosis transmembrane conductance regulator (CFTR) is unique among ion channels in that after its phosphorylation by protein kinase A (PKA), its ATP-dependent gating violates microscopic reversibility caused by the intimate involvement of ATP hydrolysis in controlling channel closure. Recent studies suggest a gating model featuring an energetic
Yeh, Han-I +2 more
openaire +2 more sources
Validation of nasospheroids to assay CFTR functionality and modulator responses in cystic fibrosis
Scientific Reports, 2021 The availability of a simple, robust and non-invasive in vitro airway model would be useful to study the functionality of the cystic fibrosis transmembrane regulator (CFTR) protein and to personalize modulator therapy for cystic fibrosis (CF) patients ...
Maite Calucho +5 more
doaj +1 more source
Evolving Cystic Fibrosis Therapy: The Good, the Sad, and the Hopeful
ChildrenCystic fibrosis (CF) is an autosomal recessive disease caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Recently developed effective CFTR modulator drugs have substantially altered the disease trajectory of ...
Dominik Funken, Hartmut Grasemann
doaj +1 more source
Physiological Reports, 2020 Introduction One method for assessing the in vitro response to CFTR‐modulating compounds is by analysis of epithelial monolayers in an Ussing chamber, where the apical and basolateral surfaces are isolated and the potential difference, short‐circuit ...
Preston E. Bratcher +4 more
doaj +1 more source
Lived experiences of individuals with cystic fibrosis on CFTR-modulators
BMC Pulmonary Medicine, 2022 Background CFTR-modulators are a category of drugs that facilitate trafficking and opening of the abnormal CFTR protein in individuals with cystic fibrosis (CF) who have certain genetic mutations.
Annelise Page +2 more
doaj +1 more source
Advanced Healthcare Materials, EarlyView.Engineered red blood cell‐derived extracellular vesicles (eRBCEVs) are synthesized via controlled microfluidic assembly from native RBC lipids, enabling tunable encapsulation of proteins, nucleic acids, nanoparticles, and viral vectors. The platform demonstrates reproducible nanoscale architecture, preserved membrane composition, and functional cargo ...
Chiranth K. Nagaraj +23 more
wiley +1 more source
Advanced Science, EarlyView.This work establishes a novel method for generating multicellular liver organoids from control and MASH donor iPSCs. The model recapitulates several disease‐specific characteristics, with MASH donor‐derived organoids showing higher susceptibility. Lipidomic profiling of MASH organoids closely resembles MASH liver biopsies.
Ekta Minocha +5 more
wiley +1 more source
Impact of CFTR modulators on exercise capacity in adolescents with cystic fibrosis
ERJ Open ResearchBackground Exercise capacity is an independent predictor of clinical worsening in cystic fibrosis (CF). There is limited evidence of the impact of cystic fibrosis transmembrane conductance regulator (CFTR) modulators on exercise capacity in children with
Molla Imaduddin Ahmed +4 more
doaj +1 more source