Introduction to Minireview Series: Development of Human Therapeutics Based on Induced Pluripotent Stem Cell (iPSC) Technology [Molecular Bases of Disease]
December 20th, 2013 by Rao, M., Gottesfeld, J.
With the advent of human induced pluripotent stem cell (iPSC) technology, it is now possible to derive patient-specific cell lines that are of great potential for both basic research and in the development of new therapeutics for human diseases. Human iPSCs not only offer unprecedented opportunities to study cellular differentiation and model human diseases but the differentiated cell types obtained from iPSCs may become therapeutics themselves. These cells can also be used in the screening of therapeutics and in toxicology assays for potential liabilities of therapeutic agents. The remarkable achievement of transcription factor reprogramming to generate iPSCs was recognized by the award of the Nobel Prize in Medicine to Shinha Yamanaka in 2012, just six years after the first publication of reprogramming methods to generate human iPSCs (1). This minireview series highlights both the promises and challenges of using iPSC technology, for disease modeling, drug screening and in the development of stem cell therapeutics.