Introduction: The revolutionary discovery of induced pluripotent stem cells (iPSCs) by Shinya Yamanaka has exposed science to new horizons. However, genetic modifications render reprogrammed cells unstable; for that reason, non-genetic modification approaches are actively under investigation. Among these, the use of small molecules is safe, and these molecules minimally affect the genome. Although iPSCs are ready for clinical trials there are many caveats hindering successful therapy, and small molecules are the best alternative to overcome those caveats.
Areas covered: Small molecules are playing an active role in generating and improving the quality of iPSCs. In this review, we will highlight the imperative role of small molecules in accelerating the successful translation of basic research into clinical use. Particularly, those ligands that replace the need for reprogramming factors will be discussed.
Expert opinion: Stem cell research is promising for harvesting medical benefits in near future. The invention of new techniques, mechanisms elucidation, and identification of novel compounds for stem cell creation has certainly established a solid foundation for regenerative medicine. This is the beginning of a new era for the cure of most disabling diseases, and small molecules will have a definite role in successful therapeutic use of iPSCs.
Keywords: Chemical biology; ciPSC; epigenetics; iPSC; patent; reprogramming; small molecule; stem cell.