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Hematological Disorders

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Human iPS Cells in Disease Modelling

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Abstract

Hematological and immunological disorders are abnormalities of the blood system. Although the elucidation of their cellular pathophysiology has been largely based on in vitro studies using patient-derived primary hematopoietic cells or animal models, these approaches have potential limitations. Induced pluripotent stem cells (iPSCs) are potential cell sources for regenerative medicine and other clinical applications, such as cell therapy, drug screening, and the investigation of disease mechanisms. The discovery of disease-associated iPSCs has led to the development of a new field of disease modeling, which can provide somatic cells that cannot be directly obtained from the patient. There have been a number of reports evaluating the use of patient-derived iPSCs to treat hematological and immunological disorders, such as bone marrow failure, hemoglobinopathy, congenital immunodeficiency, hematological malignancies, and chromosomal abnormalities. In this chapter, we review these reports and focus on the application of disease-associated iPSCs for understanding human hematological and immunological disorders while discussing the current state of hematopoietic differentiation and future perspectives.

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Acknowledgments

This work was partly supported from a grant from the Ministry of Education, Culture, Sports, Science and Technology (MEXT).

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The authors declare no conflicts of interest.

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  1. Department of Clinical Application, Center for iPS cell research and application, Kyoto University, 53 Shogoin-Kawahara, Sakyo-ku, Kyoto, 606-8507, Japan

    Megumu K. Saito & Akira Niwa

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  1. Megumu K. Saito
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  2. Akira Niwa
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Correspondence to Megumu K. Saito .

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  1. Keio University School of Medicine, Department of Cardiology, Tokyo, Tokyo, Japan

    Keiichi Fukuda

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Saito, M.K., Niwa, A. (2016). Hematological Disorders. In: Fukuda, K. (eds) Human iPS Cells in Disease Modelling. Springer, Tokyo. https://doi.org/10.1007/978-4-431-55966-5_6

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