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Disease models

Patient-specific cell culture model of cystic fibrosis (CF)

Human cell culture studies suggest in vitro-differentiated patient-derived stem cells could be useful for screening CF therapeutics. Patient-derived induced pluripotent stem (iPS) cells treated with a series of growth factors in vitro differentiated into airway epithelial cells. These cells expressed mutant cystic fibrosis transmembrane conductance regulator (CFTR) and showed lower ion flux than epithelial cells derived from healthy controls. Next steps include optimizing the differentiation procedure to improve yield and minimize generation of other cell types.

SciBX 5(41); doi:10.1038/scibx.2012.1094
Published online Oct. 18, 2012

Unpatented; licensing status not applicable

Wong, A.P. et al. Nat. Biotechnol.; published online Aug. 26, 2012;
Contact: James Ellis, The Hospital for Sick Children, Toronto, Ontario, Canada

Contact: Janet Rossant, same affiliation as above