cystic fibrosis

[European Respiratory Journal] Investigators used an unbiased RNA sequencing approach in complementary models of cystic fibrosis transmembrane conductance regulator (CFTR) silencing and blockade in human endothelial cells to characterise the changes upon CFTR impairment.

[Pharmacological Reports] Transforming growth factor-beta (TGF-β), by internalization of the epithelial sodium channel (ENaC), suppresses the anti-oxidant system, downregulates the cystic fibrosis transmembrane conductance regulator (CFTR), and activates the plasminogen activator inhibitor 1 (PAI-1) and nuclear factor-kappa-light-chain-enhancer of activated B cells (NF-kB).

[Molecular Therapy] Scientists used CRISPR/Cas9 and two adeno-associated viruses carrying the two halves of the cystic fibrosis transmembrane conductance regulator (CFTR) cDNA to sequentially insert the full CFTR cDNA along with a truncated CD19 enrichment tag in upper airway basal stem cells and human bronchial basal stem cells.

[AzurRx BioPharma, Inc.] AzurRx BioPharma, Inc. announced it has completed enrollment in its Phase II trial evaluating MS1819 in combination with the current standard of care, porcine-derived pancreatic enzyme replacement therapy (PERT), for the treatment of severe exocrine pancreatic insufficiency in patients with cystic fibrosis.

[Thorax] The authors provide a broad overview of the roles of extracellular vesicles (EV) in chronic respiratory disease. Recent advances in profiling EVs have shown their potential as biomarker candidates. Further studies have provided insight into their disease pathology, particularly in inflammatory processes across a spectrum of lung diseases.

[Scientific Reports] Investigators loaded hybrid core–shell nanoparticles (hNPs) with a 7-mer peptide nucleic acid (PNA) previously considered for its ability to modulate the post-transcriptional regulation of the cystic fibrosis transmembrane conductance regulator (CFTR) gene. They also investigated the in vitro release kinetics of hNPs and their efficacy in PNA delivery across the human epithelial airway barrier using an ex vivo model based on human primary nasal epithelial cells from CF patients.