期刊
STEM CELLS TRANSLATIONAL MEDICINE
卷 5, 期 5, 页码 572-579出版社
WILEY
DOI: 10.5966/sctm.2015-0276
关键词
Directed differentiation; Pancreatic defects of cystic fibrosis; Reprogramming; Chloride ion efflux assay
资金
- New York Stem Cell Foundation [R-103]
- DP2 [1 DP2 DK098093-01]
- Weill Cornell Medical College internal grant
- Cystic Fibrosis Foundation [CHEN15XX0]
- New York State Stem Cell Science (NYSTEM) postdoctoral fellowship
- New York State (NYSTEM) [C029156]
We established an efficient strategy to direct human pluripotent stem cells, including human embryonic stem cells (hESCs) and an induced pluripotent stem cell (iPSC) line derived from patients with cystic fibrosis, to differentiate into pancreatic ductal epithelial cells (PDECs). After purification, more than 98% of hESC-derived PDEC5 expressed functional cystic fibrosis transmembrane conductance regulator (CFTR) protein. In addition, iPSC lines were derived from a patient with CF carrying compound frameshift and mRNA splicing mutations and were differentiated to PDECs. PDECs derived from Weill Cornell cystic fibrosis (WCCF)-iPSCs showed defective expression of mature CFTR protein and impaired chloride ion channel activity, recapitulating functional defects of patients with CF at the cellular level. These studies provide a new methodology to derive pure PDECs expressing CFTR and establish a disease in a dish platform to identify drug candidates to rescue the pancreatic defects of patients with CF.
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