We recently established conditions allowing for long-term expansion of epithelial organoids
from intestine, recapitulating essential features of the in vivo tissue architecture. Here we …

Identifying subjects with cystic fibrosis (CF) who may benefit from cystic fibrosis transmembrane
conductance regulator (CFTR)–modulating drugs is time-consuming, costly, and …

Background Elexacaftor–tezacaftor–ivacaftor is a small-molecule cystic fibrosis transmembrane
conductance regulator (CFTR) modulator regimen shown to be efficacious in patients …

Adult stem cell-derived organoids are three-dimensional epithelial structures that
recapitulate fundamental aspects of their organ of origin. We describe conditions for the long-term …

In vitro drug tests using patient-derived stem cell cultures offer opportunities to individually
select efficacious treatments. Here, we provide a study that demonstrates that in vitro drug …

Abstract Information is lacking on the clinical impact of the novel coronavirus, SARS-CoV-2,
on people with cystic fibrosis (CF). Our aim was to characterise SARS-CoV-2 infection in …

Adenine base editing (ABE) enables enzymatic conversion from AT into GC base pairs. ABE
holds promise for clinical application, as it does not depend on the introduction of double-…

Rationale: Cystic fibrosis (CF) is characterized by early structural lung disease caused by
pulmonary infections. The nasopharynx of infants is a major ecological reservoir of potential …

Over the past two decades there has been considerable progress with the evaluation and
management of infants with an inconclusive diagnosis following Newborn Screening (NBS) for …

Respiratory tract infections (RTI) are more commonly caused by viral pathogens in children
than in adults. Surprisingly, little is known about antibiotic use in children as compared to …