A limiting factor for the identification of disease mechanisms and
development of new therapies has been the access to a model system/s that can
faithfully recapitulate key features of the disease and more precise clinical translations
of new treatments. Stem cells in this regard are very promising, but the ethical issues
related to totipotent embryonic stem cells and functional constraints to unipotent
somatic stem cells have led to focus on induced pluripotent stem cells to avoid both
functional and ethical constraints. The introduction of human Induced Pluripotent Stem
Cell (iPSC) technology provides a model system to replicate diseases in humans. In
this technology, human somatic cells can be “reprogrammed” by the transgene
expression of four transcription factors into stem cells called iPSC. In this chapter, it
will be discussed how iPSCs can be used for disease modelling, drug discovery and
regenerative medicine.
Keywords: Disease modelling, Embryonic Stem Cells, Induced Pluripotent Stem Cells