Journal
DISEASE MODELS & MECHANISMS
Volume 12, Issue 6, Pages -Publisher
COMPANY BIOLOGISTS LTD
DOI: 10.1242/dmm.039321
Keywords
iPSC generation; Hematopoietic differentiation; Genetic blood disorders; Monogenic disorders; Myeloid malignancies
Categories
Funding
- National Institutes of Health [R01HL121570, R01HL137219, R01HL132071, R01CA225231]
- Edward P. Evans Foundation
- New York State Stem Cell Science
- Henry and Marilyn Taub Foundation
- Alex's Lemonade Stand Foundation for Childhood Cancer
- Leukemia and Lymphoma Society Scholar award
- RUNX1 Research Program
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Induced pluripotent stem cells (iPSCs) are derived from somatic cells through a reprogramming process, which converts them to a pluripotent state, akin to that of embryonic stem cells. Over the past decade, iPSC models have found increasing applications in the study of human diseases, with blood disorders featuring prominently. Here, we discuss methodological aspects pertaining to iPSC generation, hematopoietic differentiation and gene editing, and provide an overview of uses of iPSCs in modeling the cell and gene therapy of inherited genetic blood disorders, as well as their more recent use as models of myeloid malignancies. We also discuss the strengths and limitations of iPSCs compared to model organisms and other cellular systems commonly used in hematology research.
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