Key rationale to study hematopoiesis from hPSCs
| Rationale . | Description . | References . |
|---|---|---|
| hPSCs provide an optimal model to study basic human developmental biology. | Distinct differences in hematopoietic development between humans and mice (and other species) are reflected in embryogenesis, globin gene expression, and other genes that regulate developmental pathways. | 10,27,–29 |
| hPSCs provide a model for human genetics and platform for gene therapy. | Human iPSC lines have been derived from patients with diverse genetic diseases. These cell lines can be used to define how specific mutations affect development and function of cells of interest. These also provide potential for combined cell and gene therapy to cure these diseases. hESCs can also be derived from patients with genetic disease or engineered to model gene mutations. | 30,,,,,,–37 |
| Pharmaceutical testing with hPSC-derived cells can be used to evaluate efficacy of novel therapies. | hPSCs play a growing role in drug development and testing in the future. Current efforts by industry are mainly on testing of hPSC-derived cardiomyocytes but can also expand to testing of hematopoiesis and other cell lineages. | 38,–40 |
| hPSCs can provide a source of cells for transfusion medicine. | hPSC-derived RBCs and platelets can provide an important adjunct population to that collected from donors, with reduced risk of transmissible infectious agents. | 41,,,–45 |
| hPSCs can provide a novel source for immune therapies. | NK cells, T cells, and dendritic cells have been derived from hPSCs. Novel properties of hPSCs may allow improved targeting of hPSC-derived effector lymphocytes. | 46,,,,,–52 |
| hPSCs may provide an alternative source of cells for HCT. | hPSC-derived cells may be engineered to allow beneficial properties such as drug resistance that may allow more effective administration of chemotherapy to kill tumors. Also hESC-derived HSCs may generate hematopoietic chimerism to promote engraftment of other hESC-derived lineages. | 53,,–56 |
| Rationale . | Description . | References . |
|---|---|---|
| hPSCs provide an optimal model to study basic human developmental biology. | Distinct differences in hematopoietic development between humans and mice (and other species) are reflected in embryogenesis, globin gene expression, and other genes that regulate developmental pathways. | 10,27,–29 |
| hPSCs provide a model for human genetics and platform for gene therapy. | Human iPSC lines have been derived from patients with diverse genetic diseases. These cell lines can be used to define how specific mutations affect development and function of cells of interest. These also provide potential for combined cell and gene therapy to cure these diseases. hESCs can also be derived from patients with genetic disease or engineered to model gene mutations. | 30,,,,,,–37 |
| Pharmaceutical testing with hPSC-derived cells can be used to evaluate efficacy of novel therapies. | hPSCs play a growing role in drug development and testing in the future. Current efforts by industry are mainly on testing of hPSC-derived cardiomyocytes but can also expand to testing of hematopoiesis and other cell lineages. | 38,–40 |
| hPSCs can provide a source of cells for transfusion medicine. | hPSC-derived RBCs and platelets can provide an important adjunct population to that collected from donors, with reduced risk of transmissible infectious agents. | 41,,,–45 |
| hPSCs can provide a novel source for immune therapies. | NK cells, T cells, and dendritic cells have been derived from hPSCs. Novel properties of hPSCs may allow improved targeting of hPSC-derived effector lymphocytes. | 46,,,,,–52 |
| hPSCs may provide an alternative source of cells for HCT. | hPSC-derived cells may be engineered to allow beneficial properties such as drug resistance that may allow more effective administration of chemotherapy to kill tumors. Also hESC-derived HSCs may generate hematopoietic chimerism to promote engraftment of other hESC-derived lineages. | 53,,–56 |
hPSC indicates human pluripotent stem cell; iPSC, induced pluripotent stem cell; hESC, human embryonic stem cell; RBC, red blood cell; NK, natural killer; HCT, hematopoietic cell transplantation; and HSC, hematopoietic stem cell.