The development of iPSC technology has generated a powerful new way to both define and treat diseases. iPSCs represent a paradigm shift because they now allow us to directly observe and treat relevant patient cells. In particular, they have revealed new relationships in gene expression, which have broadened and deepened our understanding of disease development in patients with sporadic disease. Progress with other technologies, such as CRISPR/Cas9, 3D organoids, and microRNA switches, will further advance the already rapid pace of iPSC-based disease modeling and therapeutic development.