GLOBE lentiviral vector

The GLOBE vector is a lentiviral vector designed for gene therapy, specifically targeting β-thalassemia. It works by genetically modifying autologous hematopoietic stem cells to introduce a functional β-globin gene, aiming to restore effective hemoglobin production in patients with β-thalassemia. This approach reduces or eliminates the need for blood transfusions in treated patients. The mechanism involves delivering the therapeutic β-globin gene into patient stem cells using a lentivirus-based delivery system (lentiviral vector). Clinical trials have shown that treatment with GLOBE-transduced CD34+ cells is well tolerated and leads to multilineage engraftment of gene-marked cells and significant reduction in transfusion requirements. The development has been conducted primarily by academic groups involved in the TIGET-BTHAL clinical trial program focused on β-thalassemia[5][6].