Making Hematopoietic Stem Cells: Backwards and Sideways

Hematopoietic stem cells (HSCs) are the functional units of bone marrow transplantation procedures. Despite wide clinical use, HSC transplantation remains a high-risk procedure and transplantation outcomes are impacted by multiple factors. One of the unmet challenges that fundamentally impact all of the current and future therapeutic uses of HSCs, is the current inability to maintain or expand HSCs ex vivo. We recently engineered a strain of mice that permits facile identification of HSCs based on single color fluorescence and have used this in small molecule screens to identify compounds capable of maintaining/ expanding HSCs ex vivo. Using this platform we have identified compounds that efficiently expand human CD34+ HSCs from bone marrow, mobilized peripheral blood, and cord blood.