Induced pluripotent stem cell technology: A window for studying the pathogenesis of acquired aplastic anemia and possible applications  Mahmoud I. Elbadry, J. Luis Espinoza, Shinji Nakao  Experimental Hematology  Volume 49, Pages 9-18 (May 2017) DOI: 10.1016/j.exphem.2016.12.011 Copyright © 2017 ISEH - International Society for Experimental Hematology Terms and Conditions

Figure 1 Conclusive roles of iPSC derivation and applications. Acquired aplastic anemia (aAA) patient-derived iPSCs can produce various HSCs with the same genetic information as the patient. These cells can then be used to clarify different strategies to study the pathophysiology of aAA using in vitro methods and animal models. Because HSPCs with 6pLOH(+) and 6pLOH(−) genotypes can be successfully generated in vitro from AA patient-derived iPSCs, these HSPCs should theoretically be useful for identifying specific antigens targeted by CTLs, as the 6pLOH(−) HSPCs, but not their 6pLOH(+) HSPC counterparts, harbor the antigens that are likely recognized by pathogenic CTLs. Also illustrated here is the possible application of autoantigen identification. Gene-corrected autologous cell therapy can be used in combination with correction mediated by ZFN or CRISPR/Cas9. iPSC-HPSCs will prove useful in regenerative medicine in the generation of patient-specific blood products and screening of effective and safe drugs, as well as in treatment of patients through cell transplantation therapy. The figure summarizes the blood cell types successfully generated using iPSCs to date and the challenges facing the application of this technology. CRISPR/Cas9 = clustered regularly interspaced short palindromic repeats; 6pLOH = copy number neutral loss of heterozygosity of the short arm of chromosome 6; PBMC = peripheral blood mononuclear cell; DCs = dendritic cells. Experimental Hematology 2017 49, 9-18DOI: (10.1016/j.exphem.2016.12.011) Copyright © 2017 ISEH - International Society for Experimental Hematology Terms and Conditions