Recent progress in human induced pluripotent stem cells (iPSCs) has opened the door to a better understanding of the biology of human diseases, especially rare disorders such as acquired aplastic anemia, in which the target hematopoietic tissues are depleted. The advent of somatic cell reprogramming has presented new routes for generating hematopoietic stem cells from patient-derived iPSCs and their differentiation into hematopoietic lineages. The purpose of this review is to discuss the recent advances in iPSC research technology and their potential applications in disease modeling for understanding the pathogenesis of bone marrow failure syndrome and the potential clinical utility of iPSC-derived cells.