Disease Modeling With Patient-Specific Induced Pluripotent Stem Cells

The development of human embryonic stem cell technology1 and the ability to differentiate these pluripotent stem cells into somatic cells, such as cardiomyocytes,2 have spawned the idea to use somatic cells generated from pluripotent stem cells as a novel type of in vitro disease model.3 The quest for pluripotent stem cell–based disease models was propelled in 2006 by the demonstration that murine somatic cells can be reprogrammed to pluripotent stem cells (termed “induced pluripotent stem cells ” [iPSCs]) that closely resemble embryonic stem cells just by forcing them to overexpress a specific cocktail of tran-scription factors.4 Barely 1 year later, this method was suc-cessfully transferred to human cells.5,6 With iPSC technology, it became feasible to take somatic cells from a patient with a heritable disorder and generate patient-specific iPSCs, which carry the disease-causing genotype. These cells can be differ