ABSTRACT
Modeling of disease phenotypes in the laboratory has long been recognized as both a means to better understand a pathological process and a tool for discovering potential treatments. The analysis of animal models exhibiting induced or naturally occurring features similar to human disease is an important preclinical activity, and the ability to genetically engineer animals, especially mice, to harbor mutations or to simulate cellular defects known to drive human disease has been a powerful tool for investigating disease mechanisms and test novel drug candidates. However, animal models, while reflective of whole-body physiology, do not truly recapitulate the human condition and often point to treatments that prove ineffective when translated to human patients. In addition, they tend to be expensive, time and labor intensive, and may pose ethical challenges. The ability to procure and culture both animal and human cells, including the use of immortalized cell lines, has been fundamental for our understanding of cell biology and has led to the development of in vitro disease models that have fueled decades of academic research and have enabled the pharmaceutical industry to discover drugs based on molecular targets or other disease-relevant defects observable in cellular models. While these approaches continue to have use, they suffer from limitations. Human cell models overcome species-related
16.1 Introduction .................................................................................................. 345 16.2 Current Uses of hPSC Disease Models ........................................................346
