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Hoffmann, R. A wiki for the life sciences where authorship matters. Nature Genetics (2008)

Complete correction of the Wiskott-Aldrich syndrome by allogeneic bone-marrow transplantation.

Two patients with the Wiskott-Aldrich syndrome had complete donor lymphoid and hematopoietic engraftment after successful allogeneic bone-marrow transplantation. One patient had had only a temporary donor T-lymphocyte graft after a previous transplantation, for which he had been prepared with cytarabine and cyclophosphamide; the patient's own T lymphocytes returned six months later. A repeat transplant, for which the patient was prepared with anti-human thymocyte serum, total-body irradiation and procarbazine, resulted in complete donor engraftment. The second patient underwent a successful transplantation after similar preparation, except that procarbazine was omitted. At 11 and five months after transplantation both had normal hematopoiesis and no evidence of graft-versus-host disease. This treatment of the Wiskott-Aldrich syndrome may be a model for the correction of other genetically determined immune and hematologic bone-marrow disorders.[1]


  1. Complete correction of the Wiskott-Aldrich syndrome by allogeneic bone-marrow transplantation. Parkman, R., Rappeport, J., Geha, R., Belli, J., Cassady, R., Levey, R., Nathan, D.G., Rosen, F.S. N. Engl. J. Med. (1978) [Pubmed]
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