Acquired aplastic anemia (AA) is considered an immune-mediated disease because approximately 70% of AA patients improve with immunosuppressive therapy. However, little is known about the inciting antigens or the mechanisms responsible for the destruction of hematopoietic stem cells by immune system attack. Recent advances in immunologic techniques have promoted our understanding of the pathogenesis of AA and have provided evidence that AA is an organ-specific T-cell-mediated disease localized in the bone marrow. Moreover, antibody screening of patients' serum with a complementary DNA library derived from hematopoietic cells has identified several proteins as candidate autoantigens in AA.