Investigating human disease using stem cell models.

Tractable and accurate disease models are essential for understanding disease pathogenesis and for developing new therapeutics. As stem cells are capable of self-renewal and differentiation, they are ideally suited both for generating these models and for obtaining the large quantities of cells required for drug development and transplantation therapies. Although proof of principle for the use of adult stem cells and embryonic stem cells in disease modelling has been established, induced pluripotent stem cells (iPSCs) have demonstrated the greatest utility for modelling human diseases. Furthermore, combining gene editing with iPSCs enables the generation of models of genetically complex disorders.