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Reprogramming cells into blood stem cells to treat leukemia

Post-Doctoral Education Grant Recipient: Kyung-Dal Choi

Faculty Sponsor: Michael Kyba

Grant Period: 2015-2016

Site: Department of Pediatrics | University of Minnesota

Click here to read Choi's Final Report

More than 150,000 individuals in the United States are diagnosed with leukemia or other blood cancers each year. While there are treatment options available, approximately 50,000 patients with blood cancer die each year.

Blood disorders are typically treated with bone marrow transplants from a donor; the transplanted bone marrow creates healthy blood cells in the patient. Unfortunately, though, this type of treatment has several issues. It's often very difficult to find a willing donor who is a close enough genetic match to the patient, and some bone marrow transplants are rejected by the body due to a lack of gene similarity. Bone marrow can also carry other diseases or genetic problems that could be transmitted to the already weak patient.

Because of these issues, scientists have been working to discover alternate types of therapy for treating blood disorders. In particular, they need another source for blood stem cells, also called hematopoietic stem cells, which will be compatible with patients. Some scientists have been using human embryonic stem cells to try developing hematopoietic stem cells, but it is a difficult procedure which isn’t very successful.

New technology allows some cell types to be converted into other types of cells through a process called reprogramming. This research will explore the possibility of making skin cells from a particular individual into hematopoietic stem cells that could then be used for treatment. Previous research has already identified three specific genes that, when inserted into skin cells, may be able to reprogram them into hematopoietic stem cells.

This project will further explore the process of reprogramming cells into hematopoietic stem cells that could survive in vivo, or within a living being, rather than just in laboratory conditions. To do this, researchers will further study the genetic regulatory mechanisms that are active in these cells and potentially explore using other cells as the starting point for reprogramming. The scientists will also test the reprogrammed cells in animals and determine whether they continue to function as normal hematopoietic stem cells in vivo.

If successful, this research may present a new treatment option that would allows a patient's own cells to be reprogrammed into hematopoietic stem cells and implanted back in their body to treat the blood disease. This would hopefully increase survival rates and help improve the quality of life for individuals who are diagnosed with leukemia and other types of blood cancer.