Modeling Disease in a Dish

Abstract

Main idea for therapeutic approaches by iPSCs at the beginning was the fact that patient-specific iPSCs provide important information for inherited human disorders because pluripotent stem cells are capable of differentiation into most, if not all cell types. This idea was deeply relied on the studies of directed differentiation of subtypes and genetically defined ESCs from animal models. Moreover, human ESC biology has been pursuing generating mutant human ESC lines as disease models since Thomson et al. derived human ESC lines in 1998. With the known disease-associated genetic loci and explicit disease phenotype, genetically modified human ESCs could help to cell replacement therapies and modeling human diseases.