Meconium ileus at birth, distal intestinal obstruction syndrome (DIOS), and constipation are
an interrelated group of intestinal obstruction syndromes with a variable severity of …

Cystic fibrosis is the most prevalent inherited disease caused by a defect in the cystic fibrosis
transmembrane conductance regulator (CFTR) gene. The impaired electrolyte homeostasis …

Single murine and human intestinal stem cells can be expanded in culture over long time
periods as genetically and phenotypically stable epithelial organoids. Increased cAMP …

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 …

Organoids are self‐organizing 3D structures grown from stem cells that recapitulate
essential aspects of organ structure and function. Here, we describe a method to establish …

Approximately 10% of all pathological mutations are nonsense mutations that are
responsible for several severe genetic diseases for which no treatment regimens are …

Background Combination treatment with the cystic fibrosis transmembrane conductance
regulator (CFTR) modulators tezacaftor (VX-661) and ivacaftor (VX-770) was designed to …

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

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 …

Background Children's physical activity patterns are characterized by short-term anaerobic
activities. Anaerobic exercise performance in children with cystic fibrosis (CF) has received …