Mesenchymal Gene Therapy

Busan, Korea -

The period of pancytopenia after hematopoietic stem cell transplantation is a significant cause of morbidity and mortality. Accordingly, growth factors, such as G-CSF have been developed clinically to reduce the period of neutropenia so as to minimize the risk of infection during the immunosuppressed state.

Since mesenchymal stem cells are known to secrete numerous hematopoietic stem cell expanding factors, as well as possessing membrane bound stimulants, researchers have previously used mesenchymal stem cells to accelerate hematopoietic stem cell engraftment.

This approach was taken a step further in a recent paper (Han et al. Cotransplantation of Cord Blood Hematopoietic Stem Cells and Culture-Expanded and GM-CSF-/SCF-Transfected Mesenchymal Stem Cells in SCID Mice. J Korean Med Sci. 2007 Apr;22(2):242-7) in which mesenchymal stem cells were transfected with GM-CSF or with Stem Cell Factor (c-kit ligand), transfered into a NOD SCID, followed by infusion of hematopoietic stem cells.

Engraftment was faster with the transfected mesenchymal stem cells as compared to the non-transfected ones.

These data demonstrate preclinical evidence that mesenchymal stem cells may be a great delivery vector for localizing hematopoietic-promoting cytokines to areas where they are needed.