Gupta, Pankaj, M.D.

HEMATOLOGY-ONCOLOGY/ CANCER RESEARCH

My laboratory is investigating the biology of human hematopoietic stem cells, specifically the role of heparan sulfate proteoglycans in the long term ex-vivo maintenance and expansion of pluripotent stem cells. We have recently identified a heparan sulfate oligosaccharide which helps maintain primitive human stem cells (LTC-IC) in-vitro. Current studies are further characterizing these proteoglycans, examining their mechanism(s) of action as well as developing stroma-free, serum-free culture conditions which will induce expansion of multipotential lympho-hematopoietic human stem cells in culture. These studies may have application for expansion of benign stem cells for autologous transplantation and for successful gene transfer into stem cells. We have identified a mechanism by which structurally abnormal heparan sulfates contribute to the pathophysiology of Hurler’s syndrome (mucopolysaccharidosis type I). We are examining if normal bone marrow-derived multi-potent adult progenitor cells (MAPC) can correct the metabolic defect and behavioral deficits in a mouse model of this disease. These studies may have implications for clinical trials of MAPC transplantation.