Abstract Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) is a chloride and
bicarbonate channel in secretory epithelia with a critical role in maintaining fluid …

As highly effective CFTR modulator therapies (HEMT) emerge, there is an unmet need to
find effective drugs for people with CF (PwCF) with ultra-rare mutations who are too few for …

Most of the 2,100 CFTR gene variants reported to date are still unknown in terms of their
disease liability in Cystic Fibrosis (CF) and their molecular and cellular mechanism that …

We present a protocol to generate organoids from air-liquid-interface (ALI)-differentiated
nasal epithelia. We detail their application as cystic fibrosis (CF) disease model in the cystic …

The plasma membrane (PM) stability of the cystic fibrosis transmembrane conductance
regulator (CFTR), the protein which when mutated causes Cystic Fibrosis (CF), relies on …

Background The forskolin-induced swelling (FIS) assay measures CFTR function on patient-
derived intestinal organoids (PDIOs) and may guide treatment selection for individuals with …

Mutations in the CFTR anion channel cause cystic fibrosis (CF) and have also been related
to higher cancer incidence. Previously we proposed that this is linked to an emerging role of …

Cystic Fibrosis (CF) is a paradigmatic disease that has led the way under multiple aspects in
the field of rare diseases. Personalized medicine for CF is the next challenge to treat all …

Cystic fibrosis (CF) is a life-threatening disease caused by variants in the cystic fibrosis
transmembrane conductance regulator (CFTR) gene. This gene encodes a transmembrane …

Mutations in the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) protein
cause Cystic Fibrosis (CF), the most common life-shortening monogenic condition in …