CFTR encodes for a chloride ion channel expressed primarily in secretory epithelia in the airways, intestine, liver and other tissues. Mutations in the CFTR gene have been identified in people suffering from Cystic Fibrosis. Here, we established a CFTR knock-in reporter cell line from a human iPSC line (MHHi006-A) using TALEN technology. The reporter enables the monitoring and optimization of the differentiation of pluripotent stem cells into CFTR expressing epithelia on a single cell level, as well as the enrichment of CFTR positive cells, which represent an excellent tool for Cystic Fibrosis disease modelling, drug screening and ultimately cellular therapies.
- Engels, L.
- Olmer, R.
- de la Roche, J.
- Gohring, G.
- Ulrich, S.
- Haller, R.
- Martin, U.
- Merkert, S.
Keywords
- Action Potentials/drug effects
- Cell Differentiation
- Cell Line
- Cellular Reprogramming
- Colforsin/pharmacology
- Cystic Fibrosis Transmembrane Conductance Regulator/*genetics
- *Gene Editing
- Humans
- Induced Pluripotent Stem Cells/*cytology/metabolism
- Karyotype
- Male
- Transcription Activator-Like Effector Nucleases/genetics
