This technology is a genetic approach for reprogramming blood progenitors into hematopoietic stem cells (HSCs), thereby enabling autologous transplantation for the treatment of blood disorders.
The ability to reprogram committed cells to a pluripotent state is an exciting avenue for medicine, particularly in the area of blood disorders, as it is thought that reprogramming could provide a method of obtaining elusive autologous hematopoietic stem cells (HSCs) for patient-specific treatment options. Despite this excitement, reprogramming has been difficult and costly to implement in practice. Thus, there is a clear need for a simplified reprogramming technique that generates transplantable HSCs from committed blood progenitors in humans.
This technology uses a single gene, BAZ2B, to reprogram human committed blood progenitors into bone marrow-engraftable hematopoietic stem progenitors. Lentiviral-mediated overexpression of this gene promotes chromatin remodeling and activates other genes that reprogram committed cells into HSCs. Through this approach, HSCs can be produced from committed progenitors in human cell lines, potentially enhancing the ability to rapidly and cost-effectively produce significant amounts of patient-specific HSCs for autologous transplantation. As such, this technology would be useful for stem cell transplantations for blood disorders, wherein non-autologous HSCs are often rejected following engraftment.
This technology has been validated with umbilical cord blood CD34+ cells.