CRISPR/cas9 editing in induced pluripotent stem cells: A way forward for treating cystic fibrosis?

Abstract

Despite drastic improvements in the treatment of cystic fibrosis (CF), there are still no curative options and severe CF remains a terminal diagnosis. With the recent advances in state-of-the-art techniques such as induced pluripotent stem cells (iPSC), stem cell expansion, differentiation, and gene editing technologies, we now have the ability to expand patient-specific cells for therapeutic application. These autologous cells can be readily genetically edited and clonally expanded with the potential for differentiation into the desired mature cell types. Such techniques hold enormous potential for therapeutic application. This chapter aims to provide an overview of the current state of iPSCs, proximal lung stem cells and gene editing techniques focusing on their potential for clinical application in CF.