Genome Editing Strategies to Cure Cystic Fibrosis

Cystic Fibrosis (CF) is a recessive genetic disorder with fatal consequences caused by over 1,700 mutations in the Cystic Fibrosis Transmembrane Regulator (CFTR) gene. We studied gene editing in patient-derived induced pluripotent stem cells (iPSCs) by correcting the most prevalent disease-causing mutation, F508del, a 3-bp deletion that prevents CFTR protein to fold properly. The challenges in edi…