My principal research interests have been the assessment of the immunological consequences of hematopoietic stem cell transplantation including both acute and chronic graft versus host disease and immune reconstitution and the use of hematopoietic stem cell transplantation to treat genetic diseases. My laboratory was the first to suggest that chronic graft versus host disease was an autoimmune disease directed at histocompatibility antigens shared by donors and recipients. The observation leaded to the assessment of the role of thymic dysfunction in the pathogenesis of chronic graft versus host disease. As a pediatric immunologist I have investigated the role of hematopoietic stem cell transplantation initially in the treatment of primary immune deficiency diseases and later the treatment of metabolic diseases, which lead to my involvement in the early gene transfer clinical trials.
Adjunct Professor, Pediatrics - Stem Cell Transplantation
Boards, Advisory Committees, Professional Organizations
Chairperson, Data Safety Monitoring Board for Cell and Gene Therapy, National Heart, Lung and Blood Institute (2002 - Present)