To date, iPSC technology has proven largely successful in modeling and recapitulating a variety of diseases in vitro,29 developing high-throughput screening (HTS) for drugs,30, 31 testing of new and existing drug toxicology32 and uncovering disease mechanisms (Figure 2).33 Combined with recent advances in genome editing technologies, iPSCs became an even more powerful tool. For example, the CRISPR/Cas9 genome editing tool has been used to establish causality of disease-causing mutations through their repair and introduction. Recently, progress has been made toward modeling genetically complex diseases, such as Parkinson’s disease, through the combination of CRISPR/Cas9, genome-wide association studies and epigenetics with iPSCs to address the role of a non-coding variant.34 In addition, CRISPR/Cas9 could be utilized to study coding variants expressed from endogenous loci and to investigate the function of single variants through isogenic controls. Hence, this technology holds promise for advancing precision medicine, patient stratification35 and cell-based therapies.36 Taken together, iPSC technology offers great potential to overcome many obstacles currently impeding progress toward understanding and treating mental illness.
