Importance of genome editing in iPS disease modelling. iPSCs can be derived from healthy (blue) and disease (orange) patients, and after differentiation into an appropriate cell type, comparison of molecular or cellular phenotypes can be made. To minimise variability due to genetic background, genome editing can be used to either correct patient-derived cells (dark blue) or to introduce putative causative lesions into cells derived from healthy individuals (purple). This leads to isogenic pairs of cell lines (purple box or orange box) that identify the true impact of the engineered change on the cellular phenotype