Early Results of a Phase I/ II Study of Gene Therapy for β-Thalassemia Major Via Transplantation of Autologous Hematopoietic Stem Cells Transduced Ex-Vivo with a Lentiviral βAT87Q -Globin Vector

Hematopoietic stem cell (HSC) gene therapy has the potential to induce globin production in the RBC lineage to diminish the need for blood transfusions in pa...