Thursday, July 11, 2013
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Intestinal organoid model
fibrosis transmembrane conductance regulator (CFTR)
An intestinal organoid
model for CFTR function could help develop compounds to treat cystic
fibrosis. Organoids were cultured from human intestinal stem cells and
treated with forskolin, which activates CFTR and
induces fluid influx and swelling. In organoids cultured from patients
carrying mutant CFTR variants, forskolin-induced swelling was lower than that
in patients carrying wild-type CFTR. In DF508 mutant CFTR-expressing
organoids, compounds that increased CFTR function also increased
forskolin-induced swelling compared with vehicle. Next steps include using
the organoids to model patient response to CFTR-targeted drugs.
Pharmaceuticals Inc.'s VX-809,
a CFTR corrector, is in Phase III trials to treat DF508
mutant cystic fibrosis (CF) in combination with the CFTR potentiator Kalydeco
Vertex markets Kalydeco to treat CF.
Published online July 11, 2013
Patent application filed;
available for licensing
Dekkers, J.F. et al.
Nat. Med.; published online June 2, 2013;
Contact: Jeffrey M. Beekman, University Medical Center
Utrecht, Utrecht, the Netherlands
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