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New therapies are turning cystic fibrosis, an inherited disorder that causes a vicious cycle of mucus build-up, infection and inflammation, into a more manageable condition that people can survive into adulthood.
Defective CFTR protein, responsible for Cystic Fibrosis (CF), is highly expressed in pancreatic ductal epithelial cells (PDECs) but their impact on insulin secreting pancreatic islets is not fully understood. Here the authors develop a non-CF and CF patient derived pancreas-on-a-chip model to study how CF affects insulin secretion.
Cystic fibrosis is caused by mutations in the CFTR chloride channel. Here, the authors develop a gene therapy approach using the programmable nuclease AsCas12a to correct a splicing mutation in CFTR, and show efficient repair of the mutation and recovery of CFTR function in patient-derived organoids and airway epithelial cells.
The leading cause of cystic fibrosis is the deletion of phenylalanine 508 (F508del) in the first nucleotide-binding domain (NBD1) of the cystic fibrosis transmembrane conductance regulator (CFTR). Here authors we develop nanobodies targeting NBD1 of human CFTR and demonstrate their ability to stabilize both isolated NBD1 and full-length protein.
Personalized approaches to diagnosis and treatment monitoring could improve the management of cystic fibrosis patients. Here the authors show that multiscale differential dynamic microscopy can assess changes in cilia beating dynamics and coordination in patient-derived airway epithelial cells, in response to different CFTR-modulating drugs, in a patient-specific manner.
Pseudomonas aeruginosa displays substantial genetic diversification across sub-compartments in cystic fibrosis (CF) lungs. Here, Kordes et al. show that, despite genetic variation, the ex vivo transcriptional profiles of P. aeruginosa populations are similar across five different areas in an explanted CF lung.
Innate lymphoid cells (ILCs) play critical immunological roles including immune surveillance at mucosal sites. Here the authors show that during nasal inflammation pathogen-induced cytokine production guides the differentiation of ILCs.
How genetic adaptation and phenotypic acclimation are interrelated and allow Pseudomonas aeruginosa to persist in cystic fibrosis lungs is poorly understood. Here, Rossi et al. use high-resolution transcriptomics on expectorates to link phenotypic conservation to ecological flexibility and persistence.
Mutations in gene pmrB are found in Pseudomonas aeruginosa isolates from cystic fibrosis patients. Here, Bricio-Moreno et al. show in a mouse model of respiratory infection that the mutations enhance bacterial adherence to epithelial cells and resistance to lysozyme, but also increase antibiotic susceptibility.
In cystic fibrosis, ion-transport abnormalities cause problems in many organs. A small molecule that forms cell-membrane pores allowing ion transport shows therapeutic promise in human cells and a model of the disease.
Amphotericin B forms nonselective transmembrane ion channels, and restores host defences of cystic fibrosis airway epithelia independently of the regulatory function of cystic fibrosis transmembrane conductance.
A nanoparticle self-assembled by a multi-modular peptide and an amphiphilic poloxamine serves as a non-viral gene delivery agent for the long-term expression of CFTR in cell cultures and in animal models of cystic fibrosis.