• Ph.D., University of Liverpool, 1988

Research Interests: The lab is focused on the therapeutic use of transplantation for inherited and acquired disorders. Our aim is to investigate stem cell therapy in murine models with defects in their hematopoietic and immune systems. The major project in the lab is the potential treatment of b-thalassemia which is one of the most common inherited disorders in man and has an increasingly global distribution. Current conventional therapies for this debilitating hemoglobinopathy are complicated, expensive, and are not curative. Hematopoietic stem cell (HSC) transplantation may cure b-thalassemia, but transplant-related morbidity and mortality is substantial. Additionally, stem cell transplantation is limited by the lack of suitable histocompatible stem cell donors. We are trying to pursue the opportunity to evalute in utero therapy for hemoglobinopathies, immunodeficiency disorders and metabolic diseases. Transplantation in utero has the potential to increase the donor pool by circumventing the immunological barriers associated with post-natal transplantation of mismatched cells, and would also allow for treatment before the onset of serious pathological episodes.Also of special interest are storage disorders, such as Krabbe disease (galactosylceramidase deficiency), that have a significant CNS component that allow for the investigation of the interaction of hematopoietic/immune systems with the CNS. We are examining approaches such as the repopulation of the CNS microglia after hematopoietic stem cell transplant and the potential of neural stem cell transplantation to repair areas of demyelination, and as a potential source of normal enzyme.