Differentiation of human embryonic and induced pluripotent stem cells into CFTR expressing cholangiocytes for drug screening and disease modeling

Introduction: Cystic fibrosis (CF) is the most common lethal monogenic recessive disease in the caucasian population. While the majority of patients die of respiratory failure, hepatobiliary disease is the third leading cause of death in CF. Over 1900 mutations are known so far in cystic fibrosis transmembrane conductance regulator (CFTR) gene, but the most common is the ΔF508 mutation, resulting in enhanced degradation of the CFTR protein. Currently approved CFTR modulators are only available for a minority of CF patients. In addition, these modulators possess varying potentials. Therefore finding new modulator…