Modeling Cystic Fibrosis in vitro: A new possible platform for patient customised drug screening and ex-vivo gene therapy?

Cystic fibrosis (CF) is the most common lethal monogenic recessive disease in the caucasian population. Over 1900 mutations are known in the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) gene, but the most common mutation is the F508del resulting in misfolding of the CFTR protein. Until today, classical gene therapy trials have not been successful in CF patients, and currently approved CFTR modulators are only effective in a minority of CF patients. In contrast, stem cell-based approaches offer new perspectives for diseases like CF. Besides their almost unlimited proliferation and differentiation po…