Use of iPS cells as a disease model and therapeutic approach

M. Esther Gallardo

During the last decade, the knowledge about the molecular basis of the genetic disorders has increased considerably. However, the molecular mechanisms underlying a genetic defect, or the development of new therapies is being much slower. This is due, in part, to the lack of suitable disease models. In 2006, the Japanese scientist Shinya Yamanaka showed that it is possible to convert adult cells into a pluripotent stem cell-like state. These cells, named induced pluripotent stem cells (iPS), are similar to embryonic stem cells (ES) and are considered a very promising tool in the field of regenerative medicine. iPS cells, like ES cells, are self-renewablel and pluripotent. Nevertheless, because iPS cells are obtained in vitro from adults cells, they would not have the ethical and legal problems associated to the use of ES cells. Besides, due to the fact that they are generated from the patient, the likelihood of rejection in autologous therapies is much lower. The purpose of this chapter is to discuss what the applications of iPS cells that turn them into a hopeful tool to delve into the physiopathogenic mechanisms behind diseases as well as the quest for new therapies against them.