Human blood stem cell expansion: Empowering new technology for stem cell medicine

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Blood stem cells are the fundamental component of regenerative medicine applications involving the blood and immune system, including bone marrow transplantation and gene therapy applications. Despite significant efforts and investment, researchers have largely failed to maintain fully functional blood stem cells for substantial periods of time outside the body. As a result, the limiting factor for cell and gene therapies is often obtaining a sufficient number of blood stem cells to seed the production of mature cells in vitro (e.g., red blood cells, platelets) or to lead to successful longterm bone marrow transplantation (e.g., gene therapy).Our recent advances in mouse blood stem cell expansion put us on the cusp of breaking through a decades-old barrier and this novel cell expansion system urgently requires characterisation and translation to human HSCs. Our proposal brings together leading UK and Japanese researchers in the molecular and functional characterisation of mouse and human blood stem cells in order to leverage their complementary expertise to translate this technology to the human system and explore potential clinical applications.