Year 3

Our lab studies in utero hematopoietic stem cell transplantation as a way novel strategy to treat fetuses with congenital stem cell disorders. This method can potentially allow us to transplant genetically foreign stem cells without rejection by the immune system. In our previous experiments, we have determined that the mother’s immune system can be a barrier to success but the fetal immune system does not reject the transplanted cells. In these experiments, we first used a mouse model and performed a detailed analysis of the fetal host immune response to transplantation to understand why rejection does not occur. We also analyzed human maternal and cord blood samples to understand human fetal immune maturation, to determine whether clinical applications will involve any immune response from the fetus or the mother. Our results are an exciting preclinical platform for considering in utero transplantation for fetuses with disorders such as hemoglobinopathies.