ABSTRACT

This chapter provides an overview on the history of stem cell science and reviews current methods and shortcomings in the generation of pluripotent stem cells and their application in pharmaceutical and clinical settings. Pluripotent stem cells are a transient cell population during embryonic development. They are unique in (i) exhibiting an unlimited self-renewal potential in vitro and (ii) generating all cells and tissue types of the adult organism. Therefore, they hold great promise for regenerative medicine and patient-specific therapies. Pluripotent stem cells isolated from the human embryo, referred to as embryonic stem cells (ES cells), have raised ethical controversies and legal restrictions. The discovery that fully developed somatic cells can be reprogrammed into pluripotent stem cells, referred to as induced pluripotent stem cells (iPSCs), revolutionized stem cell research and therapies. Today, iPSCs and their derivatives are used to (i) model and study diseases in a dish, and (ii) screen for new drugs, and test their efficacy and safety. In addition, initial iPSC-derived cell replacement therapies are in phase I and II clinical trials.