Non-myeloablative conditioning with busulfan before hematopoietic stem cell transplantation leads to phenotypic correction of murine Bernard-Soulier syndrome.

Abstract excerpt

BACKGROUND: Bernard-Soulier syndrome (BSS) is an inherited bleeding disorder characterized by macrothrombocytopenia. Platelet transfusion is used for the management of bleeding, but repeated transfusion often results in alloimmunization. We have recently shown phenotypic correction of murine BSS (GPIbα(null) ) using lethal radiation conditioning followed by hematopoietic lentivirus-mediated gene transfer....

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