Human Gene Therapy for The Inherited Platelet Bleeding Disorder Glanzmann Thrombasthenia

Glanzmann Thrombasthenia (GT) results from integrin αIIbß3 defects that prevent platelet aggregation causing lifelong mucocutaneous bleeding leading to morbidity and mortality. Uncontrolled bleeding is commonly treated with platelet transfusions, antifibrinolytics, and rFVIIa. Although, there is no adequate cure for GT. Our previous preclinical studies showed proof-of-principle that hematopoietic stem cells (HSC) modified with transgenes encoding αIIbß3 provide safe and effective long-term hemostasis in GT animal models. This suggests feasibility for genetic therapy for GT patients.