… Rescue of CFTR function with modulators measured in colon organoids can be … cystic
fibrosis. Organoids are an effective model to bring treatment to patients with CF carrying rare CFTR …

… Cystic fibrosis transmembrane conductance regulator (CFTR) is a unique member of the …
Mutations in CFTR cause cystic fibrosis, the most common genetic disease in people of …

… recent advances in CFTR folding, function and pharmacology. We … of CFTR folding, which
provide new insight into CFTR assembly, its perturbation by CF mutations and rescue by CFTR …

… of cystic fibrosis, and the development of therapies targeting defects in the cystic fibrosis
transmembrane conductance regulator (CFTR… changing landscape of cystic fibrosis care and the …

… correction of the CFTR gene. In this review we will discuss different strategies to restore
CFTR function via gene editing correction of CFTR mutations or enhanced CFTR expression, …

… CFTR (cystic fibrosis … of CFTR function in patients with cystic fibrosis (CF) with one or two
copies of the common F508del mutation. However, the extent of restoration of CFTR function …

… The development of alternative approaches to restoring cystic fibrosis transmembrane
conductance regulator (CFTR) function is needed for those with ineligible mutations. …

… correlations between CFTR genotype function and clinical features of cystic fibrosis. … low
CFTR function could benefit the most from modulator therapies, even if the increase in function …

… in addition to clinical symptoms or evidence of CFTR dysfunction.4 The CF Foundation … the
first choice for assessment of CFTR function. Loss of CFTR function results in failure to resorb …

… This assay provides simultaneous measurements of both CFTR function and CFTR … a
HS-YFP fused to the intracellular N-terminal of CFTR [Citation50]. The mCherry expression allows …