Figure 2.

Interrogation of AD‐associated gene mutations using iPSC technology and CRISPR‐Cas9 gene editing to generate isogenic (ie, same genetic background) control cell lines. iPSC cells derived from a patient affected by familial AD (FAD) carry the pathogenic mutation, which is corrected by CRISPR‐Cas9. The mutation corrected iPSCs are used to model disease development during differentiation into specific cell types of the brain (eg, neurons). Introduction of the pathogenic mutation in iPSC cells derived from a healthy patient by CRISPR‐Cas9 may be applied to demonstrate recapitulation of disease pathogenesis in vitro when compared to the isogenic controls. The CRISPR‐Cas9 platform enables the study of gene interaction networks and environmental conditions in AD.