Abstract

One of the major challenges facing modern medicine in developing new treatments is the lack of understanding of the biological and molecular processes underlying chronic human diseases with mortality or disability as the primary outcome. The relative simplicity with which human-induced pluripotent stem cells (hiPSCs) can now be generated from human cells has changed the way we study such diseases. hiPSCs can be generated from a number of different human somatic cells, including lymphoblastoids, the subject of this chapter. Lymphoblastoid-derived hiPSCs are similar to those generated from fibroblasts. They exhibit normal pluripotency characteristics, are Epstein-Barr virus virus-free, readily differentiate into three germ layer cell types, maintain their genotype, and can be differentiated into cardiomyocytes, neurons, myoblasts, and hepatocytes. hiPSCs provide an unlimited source of patient-specific cells carrying the same genetic background and can be used to derive specific cell types relevant to the diseased tissue. One of the remarkable properties of hiPSCs is that they can be differentiated to virtually any cell type. hiPSC technology provides an unparalleled method for mimicking diseases in a cell culture dish, providing a strong foundation for deciphering the underlying disease machinery, transplanting cells, screening drugs, and conducting clinical trials that could lead to exciting breakthroughs in medical research.

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