Ex vivo fucosylation of stem cells to improve engraftment: WO2004094619

Abstract

Background: The application is in the field of haematopoietic stem cell (HSC) and umbilical cord blood (CB) transplantation.Objective: It aims at determining an enzymatic procedure to improve the engraftment of CB stem cells to the bone marrow (BM) as well as the rate of success of CB transplantation.Methods: Human CB stem cells were treated with α1 – 3 fucosyltransferase VI (FTVI) for 30 min ex vivo prior to transplantation. Human CB stem cells were administered intravenously in irradiated nonobese diabetic/severe combined immune deficiency (NOD/SCID) mice.Results: Treatment of CB stem cells with FTVI improves their engraftment in the BM of NOD/SCID mice.Conclusion: Treatment of CB stem cells with FTVI, ex vivo prior to transplantation, may reduce the delay of engraftment of CB stem cells to the BM and the risk of early infections associated with CB transplants, particularly in adults. It may improve the outcome and rate of success of CB transplantation. The application claims the use of fucosyltransferase for improving the engraftment of CB HSCs to the BM for the treatment of haematologic diseases. The procedure may be used to improve the engraftment of HSCs from other sources and other types of stem cells, on transplantation, particularly when administered intravenously.