Laboratory Research

The Pediatric BMT Laboratory has several research projects involving various aspects of bone marrow stem cell transplantation. Since 1982, one segment of the laboratory has been involved in processing bone marrow cells to remove T cells in order to allow parents and other partially mismatched relatives to donate for a transplant without developing fatal graft versus host disease. Various techniques and approaches for T cell depletion and stem cell enrichment have been developed and pioneered in the BMT laboratory. These techniques have also been applied to the development of animal models for transplanting bone marrow stem cells into fetal recipients. This in utero transplant model has helped scientists and clinicians understand the complexities of engraftment in the fetus and the mechanisms that allow the recipient to accept the donor cells without the need for preparative chemotherapy.

Gene cloning involves one of the most severe types of inherited immunodeficiency diseases (Severe Combined Immunodeficiency Disease, SCID), which occurs in some Native American Indian populations. The large majority of these patients have been treated at UCSF with bone marrow transplants. Scientists in the BMT Laboratory have identified and cloned the "SCIDA" gene on human chromosome 10 and have found the unique genetic mutation, which results in this disorder. They are currently working on understanding the function and role of this gene in the development of T and B lymphocytes. To this end, they have recently engineered a mouse model of SCIDA which they will use to develop more optimal therapeutic approaches for SCIDA and other severe primary immunodeficiencies as well as possibly treating disorders that involve immune dysregulation such as autoimmune diseases and cancer.