Prospects and challenges of induced pluripotent stem cells as a source of hematopoietic stem cells


Harald M.M. Mikkers, Department of Molecular Cell Biology, Leiden University Medical Center, Postal zone S1P, PO Box 9600, 2300RC Leiden, the Netherlands.


Many life-threatening hematological diseases are now treated by bone marrow transplantations, i.e., infusion of hematopoietic stem cells (HSCs). HSC transplantations are a valid option for the treatment of a variety of metabolic disorders, and even for solid tumors and some refractory severe autoimmune diseases. Unfortunately, the frequency and outcome of HSC transplantations are limited by a shortage of suitable donors. Induced pluripotent stem cells (iPSCs)—somatic cells that have acquired pluripotent stem cell characteristics by the ectopic expression of pluripotency-inducing factors—have been proposed as an alternative source of HSCs. Possible applications include cells of autologous, of autologous and genetically modified, or of allogeneic origin. Here, we provide a perspective on the distinct opportunities of iPSCs and discuss the challenges that lie ahead.