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Acquired aplastic anemia (AA) is a condition of bone marrow failure (BMF) characterized by blood pancytopenia and BM hypoplasia. In most cases, AA is an immune-mediated disorder with destruction of hematopoietic stem and progenitor cells by T cells. There is increasing evidence that CD4+ T effector cells that produce high levels of IFN- are associated with AA in patients and experimental AA mice. IFN- displays potent effects on suppressing hematopoiesis. Immunosuppressive therapy with antithymocyte globulin in combination with cyclosporin A (CsA) can induce a hematologic response in about two-thirds of AA patients. However, relapse occurs in up to 35% of AA patients when CsA is withdrawn at 6 months. Allogeneic BM transplantation (BMT) has significantly improved the survival of AA. However, graft-versus-host disease (GVHD) remains a major barrier tothe success of the procedure. Novel approaches are needed to improve the outcomes of AA treatment.