Blood and Immune Cells
A major impediment to the therapeutic use of somatic hematopoietic stem cells is the development of graft-versus-host disease due to donor-recipient mismatches. Improved methods for expanding human embryonic stem cells and deriving highly purified populations of hematopoietic stem cells that will not initiate immunologic reactions against host tissues is a major challenge for the field. Moreover, developing methods for efficiently differentiating human embryonic stem cells into functional platelets and erythrocytes in vitro would be a major advance over the current practice of transfusing blood products from one individual to another.
Another major area of interest is the use of human embryonic stem cells and hematopoietic stem cells to correct hereditary disorders of the hematologic system, such as sickle cell disease, thalassemia, and immunodeficiency disorders. Human embryonic stem cells are an important model system to test strategies to correct these inherited disorders and may ultimately prove to be the optimal tissue for transplantation. Finally, pioneering studies of “leukemia stem cells” established the principle that rare populations of cells within cancers are required to maintain the malignant phenotype. Characterizing the biological and functional characteristics of normal and leukemic stem cells has enormous potential for developing more effective and less toxic treatments for leukemia, lymphoma, and other clonal diseases of hematopoietic cells. These problems are increasing in importance as the population ages.
This program within the Institute for Regeneration Medicine closely interacts with the Program in Hematopoietic Malignancies in the UCSF Comprehensive Cancer Center.
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