Hematopoietic Stem-Cell Transplantation for the Treatment of Severe Combined Immunodeficiency

Severe combined immunodeficiency is a rare, fatal syndrome that can be due to a variety of genetic abnormalities causing profound deficiencies of lymphocytes. 1 – 4 Shortly after the discovery of the HLA system in 1968, 5 , 6 immune function was corrected in an infant with severe combined immunodeficiency by the transplantation of bone marrow from his HLA-identical sister. 7 Over the following decade, however, lethal graft-versus-host disease (GVHD) was a major problem when marrow from HLA-mismatched donors was transplanted. 1 In the late 1970s, studies in rats 8 and mice 9 revealed that allogeneic marrow or spleen cells that were depleted of T cells rescued the . . .