Cystic Fibrosis Research Advances

In Utero CFTR Modulation Alleviates Disease in G551D Cystic Fibrosis Pigs

This paper shows that in utero ivacaftor treatment rescues multiple organ defects in CFTR-G551D pigs, improving pancreatic, gallbladder, and vas deferens function.

Divergent Neurobehavioral Effects of CFTR Modulators Elexacaftor and Ivacaftor in Mice

This study examines the neurobehavioral effects of CFTR modulators elexacaftor and ivacaftor in mice, finding anxiety-like and depressive-like behaviors respectively.

Influence of Disease-Causing Mutations and Ivacaftor on the Dynamics of the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) Protein

This study investigates how disease-causing mutations and ivacaftor affect CFTR protein dynamics using molecular simulations and cryo-EM data.

Effects of Pseudomonas aeruginosa pyocyanin and 1-hydroxyphenazine on intracellular calcium, mitochondrial function, and viability in human nasal epithelial cells

This study investigates the effects of pyocyanin and 1-hydroxyphenazine on calcium signaling, mitochondrial function, and cell viability in nasal epithelial cells and cancer cell lines.

Podoplanin expression identifies human airway basal cells with higher progenitor cell potential

This study identifies podoplanin (PDPN) as a marker for human airway basal cells with high progenitor potential, enabling efficient sorting of airway stem cells.

Respiratory viral infection is associated with increased Pseudomonas abundance in cystic fibrosis airways

This study finds that respiratory viral infections increase Pseudomonas abundance in cystic fibrosis airways, with persistent microbial changes and associations with pro-inflammatory cytokines.

Evaluating an In Vivo Oxidative Stress sensor in cystic fibrosis rat epithelial tissues

The paper evaluates an in vivo oxidative stress sensor in CF rat tissues, finding elevated redox fluctuations in Phe508del rats compared to wildtype.

Mechanistic insights into CFTR function from molecular dynamics analysis of electrostatic interactions

This study uses molecular dynamics simulations to analyze electrostatic interactions in CFTR, revealing their role in stabilizing structure and enabling ion conduction.

Comparative Single-Cell Transcriptomics Uncovers Shared and Distinct Molecular Signatures in Cystic Fibrosis and Primary Ciliary Dyskinesia

This study compares single-cell transcriptomes of Cystic Fibrosis and Primary Ciliary Dyskinesia to identify distinct molecular signatures and pathways.

Machine Learning Analysis of Cilia-Driven Particle Transport Distinguishes Primary Ciliary Dyskinesia Cilia from Normal Cilia

The paper uses machine learning to analyze cilia-driven particle transport and distinguishes PCD cilia from normal cilia with high accuracy.

Molecular prosthetics and CFTR modulators additively increase secretory HCO- flux in cystic fibrosis airway epithelia

This study shows that combining CFTR modulators with amphotericin B increases HCO3- secretion and airway surface liquid pH in CF airway epithelia more than either treatment alone.

Verapamil limits inflammation by restoring VGCC-driven epithelial Ca2+ in models of cystic fibrosis

This study shows verapamil reduces inflammation in cystic fibrosis by normalizing epithelial calcium signaling and oxidative stress in zebrafish and human cells.

Morpho-functional timeline of progressive cystic fibrosis pancreatic exocrine and endocrine pathology derived from semi-quantitative scoring and AI-driven quantitativeimage analysis

This study uses histopathological scoring and AI analysis to map the progression of pancreatic changes in cystic fibrosis across age, identifying key fibrotic and fatty tissue transformations linked to exocrine and endocrine dysfunction.

Correcting mutant CFTR with a stabilizing nanobody reveals a novel active conformation of the channel

This study uses a nanobody to stabilize mutant CFTR, revealing a novel active conformation and improving its function in cystic fibrosis.

Profiling Bile Acids in the Stools of Humans and Animal Models of Cystic Fibrosis

This study profiles bile acids in stool from children with and without cystic fibrosis, revealing differences in BA species and microbial metabolism, and highlights the ferret model's suitability for BA research in CF.

Molecular prosthetics for CFTR designed for anion selectivity outperform amphotericin B in cultured cystic fibrosis airway epithelia

The paper shows that modified amphotericin B derivatives improve ASL pH in CF airway epithelia better than amphotericin B itself.

Elexacaftor/tezacaftor/ivacaftor (Trikafta(R)) does not affect the growth of Aspergillus fumigatus in vitro

This study shows that CFTR modulators do not affect the growth of Aspergillus fumigatus in vitro, despite reducing its colonization in patients.

A stem cell-based platform for functional analysis of genetic variants in lung disease

This study develops an iPSC-based platform to functionally analyze genetic variants in lung disease, demonstrating its application in diagnosing a variant of uncertain significance in primary ciliary dyskinesia.

Gene-Corrected Basal Cells Restore CFTR In Vitro; Transplants Regenerate Epithelium in a Preclinical Sinus Model

Proteomic and structural comparison between cilia from primary ciliary dyskinesia patients with a DNAH5 defect

The paper compares proteomic and structural differences in cilia from PCD patients with DNAH5 mutations, showing patient-specific reductions in ciliary components and structural changes in the axoneme.

Cilia dynamics creates a dynamic barrier to penetration of the periciliary layer in human airway epithelia

This study develops an experimental method to measure fluid flow in the periciliary layer of human airway epithelia, revealing cilia-driven fluid expulsion and its role in pathogen barrier formation.

Photoaffinity ligand of Cystic Fibrosis corrector VX-445 identifies SCCPDH as an off-target

The paper identifies SCCPDH as an off-target of VX-445 using a photoaffinity ligand and chemoproteomics, showing metabolic dysregulation and potential clinical implications.

A chronic Pseudomonas aeruginosa mouse lung infection modeling the pathophysiology and inflammation of human cystic fibrosis

This paper develops a chronic Pseudomonas aeruginosa lung infection model in mice that mimics human cystic fibrosis (CF) pathophysiology and inflammation, showing similar lung function defects and immune responses.

CFTR mutation leads to intrinsic dysfunction in neutrophils from people with Cystic Fibrosis

This study shows that CFTR modulators restore antimicrobial functions in neutrophils from people with cystic fibrosis by correcting CFTR trafficking and chloride efflux.

ENaC contributes to macrophage dysfunction in cystic fibrosis

This study shows ENaC is overexpressed in CF macrophages and contributes to their dysfunction, with ENaC inhibition improving immune function and reducing inflammation.

Trikafta rescues F508del-CFTR by tightening specific phosphorylation-dependent interdomain interactions

This paper investigates how Trikafta corrects F508del-CFTR by altering phosphorylation-dependent interactions between transmembrane and nucleotide-binding domains, stabilizing NBD1 above body temperature.

In vitro inhibition of the CFTR ion channel in the cervix thickens cervical mucus

Inhibiting CFTR in endocervical cells leads to dehydrated, thickened mucus production in vitro.

Prevalence, Production and the Role of Staphylococcus aureus Superantigens in Cystic Fibrosis Lung Disease.

This study investigates the prevalence and role of Staphylococcus aureus superantigens in CF lung disease, showing their ability to induce inflammatory responses in mouse models.

CFTR High Expresser BEST4+ cells are pH-sensing neuropod cells: new implications for intestinal physiology and Cystic Fibrosis disease

The paper identifies CFTR High Expresser cells in rat small intestine as pH-sensing neuropod cells and links them to CF disease pathogenesis.

Proteostasis Landscapes of Cystic Fibrosis Variants Reveals Drug Response Vulnerability

The paper uses proteomics and computational modeling to analyze CFTR variants, identifying structural vulnerabilities in NBD1 that limit corrector efficacy and suggesting secondary mutations as a therapeutic strategy.