Hematopoietic stem cells (HSCs) are responsible for producing all blood and immune cells in the body and have been used for decades to treat diseases such as leukemia and other blood disorders. However, a major limitation of this therapy is the scarcity of compatible donors and the difficulty in expanding HSCs outside the body. Our project, HSC-reNEW, aims to tackle this challenge by understanding how these stem cells naturally develop in the human embryo, with the ultimate goal of reproducing that process in the lab.
To achieve this, we study human pluripotent stem cells (hPSCs)—a type of cell that can develop into any tissue in the body—as a model for how blood cells are formed during early development. By decoding the principle guiding these stem cells to become blood-forming HSCs, we aim to produce transplantable blood stem cells entirely in the lab. If successful, this would remove the dependence on donors, providing a renewable and patient-specific source of stem cells. In addition, we will activate the same principle in existing primary HSCs in the attempt to expand them - an approach that could dramatically improve outcomes and reduce risks associated with transplants.
Our research tackles three main questions:
1. How do blood cells first emerge during development?
2. Can we recreate this process to make real HSCs from hPSCs?
3. Can we expand adult HSCs in the lab to treat more patients?
The answers to these questions could revolutionize how we treat genetic blood diseases, childhood bone marrow failure syndromes, and leukemia. As such, through this work, HSC-reNEW is positioned to address a critical unmet need in medicine, opening the door to safer, more accessible, and more personalized treatments for a range of life-threatening diseases.
