Figure 1
Figure 1. Therapeutic potential of hPSCs for blood diseases. iPSCs can be potentially used to treat patients with monogenic genetic blood diseases such as sickle cell anemia, β-thalassemia, Fanconi anemia, or SCID (upper panel). Autologous skin or blood cells from these patients can be reprogrammed into iPSCs. The defective gene in iPSCs can be repaired using homologous recombination. De novo generation of HSCs from gene-corrected iPSCs would provide immunologically matched cells for bone marrow transplantation. For cancer therapy, autologous iPSCs could be generated from skin fibroblasts or other somatic cells lacking leukemia mutation and used to generate HSCs for bone marrow transplantation as well as immune cells to induce an anti-leukemia immune response (lower panel). Professional illustration by Paulette Dennis.

Therapeutic potential of hPSCs for blood diseases. iPSCs can be potentially used to treat patients with monogenic genetic blood diseases such as sickle cell anemia, β-thalassemia, Fanconi anemia, or SCID (upper panel). Autologous skin or blood cells from these patients can be reprogrammed into iPSCs. The defective gene in iPSCs can be repaired using homologous recombination. De novo generation of HSCs from gene-corrected iPSCs would provide immunologically matched cells for bone marrow transplantation. For cancer therapy, autologous iPSCs could be generated from skin fibroblasts or other somatic cells lacking leukemia mutation and used to generate HSCs for bone marrow transplantation as well as immune cells to induce an anti-leukemia immune response (lower panel). Professional illustration by Paulette Dennis.

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