Dr. Kiwon Lee, Research Associate in the laboratory of Dr. Mortimer Poncz, Chief of the Division of Hematology at Children's Hospital of Philadelphia, presented his latest RUNX1 research findings in our first RRP Research Webinar of 2022.

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.