Evolving Paradigms in Hematology: Hematopoietic Stem Cell Culture Comes of Age

Hematopoietic stem cells (HSCs) are a rare but critical cell population in hematopoietic system health, pathology, and therapy1. This self-renewing multipotent stem cell population primarily resides in the bone marrow, where they sustain life-long blood production. HSCs are now understood to be a common origin of hematological dysfunction, with acquisition of genetic mutations in HSCs being an initiating event for myeloid leukemias and myelodysplastic syndromes. Mutant HSCs also drive clonal hematopoiesis, which represents a risk factor for a range of hematological and non-hematological pathologies2. Therapeutically, allogeneic and autologous stem cell transplantation rely on the regenerative potential of HSCs and represent a mainstay treatment for many of the most serious hematological diseases. HSC gene therapies also leverage this regenerative capacity, with gene correction HSC products now approved for several hematological disorders.

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doi.org/10.17863/cam.134290