What inherited genetic diseases are potentially treatable with stem cells?
There are a large number of inherited genetic diseases (like sickle cell anemia) that can now be accurately diagnosed early in fetal life by examining fetal DNA obtained by chorionic villus sampling or amniocentesis. In most cases the testing is done because the disease has been identified to run in the family. Some of these diseases are treated after birth by bone marrow transplantation. However, successful bone marrow transplantation after birth is limited by lack of donor stem cells, damage already done to the baby by the disease, rejection of the donor cells by the baby’s immune system, and, in some cases, rejection of the baby’s tissue by the donor cells (graft-versus-host disease or GVHD). Many of the problems associated with transplantation of stem cells after birth are related to immune rejection. Transplantation before birth into a fetus whose immune system is not fully developed may overcome these problems. The strategy of transplanting normal stem cells into a fetus with a s
