Development of a differentiation model for genetic defects using human CD34+ hematopoietic stem cells (HSC) and induced pluripotent stem cells (iPSCs)
Investigation of genetic disorders related to RUNX1 (Runt-related transcription factor-1) deficiency
Mechanistic study of leukemia development induced by abnormal differentiation of hematopoietic stem cells and development of targeted therapies
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The focus of Dr. Lee’s research has been on developing clinically relevant model systems for RUNX1 haploinsufficiency. Specifically, he and his colleagues have generated RUNX1-deficient human CD34+ progenitor cells that when differentiated in vitro into megakaryocytes can be infused into immunocompromised mice.
The infused RUNX1-deficient megakaryocytes consistently produce both fewer platelet numbers and platelets with reduced function. Using this novel system and another in vivo platelet function model system, Dr. Lee shows how inhibiting transforming growth factor beta pathways can rescue both platelet release and platelet function.
The RUNX1 Research Program (RRP) is a registered 501(c)(3) charitable organization and the only group worldwide committed to leading advocacy and advancing research for families impacted by RUNX1 familial platelet disorder (RUNX1-FPD).
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