To accomplish this goal, a fragment of the integrin αIIb (ITGA2B) gene promoter known to drive megakaryocyte-specific gene transcription17 was employed for ectopic expression of human B-domain-deleted Factor VIII (BDDFVIII) because megakaryocyte-targeted expression of ITGA2B and integrin β3 (ITGB3) genes was previously shown to be useful for hematopoietic stem cell gene therapy leading to correction of platelet function in dogs and mice affected with the inherited bleeding disorder Glanzmann thrombasthenia (GT)18, 19. The gene discussed is ITGA2B; the disease is Glanzmann thrombasthenia 1.