lentiviral vector (anti-sickling β-globin)

Lentiviral vector (anti-sickling β-globin) refers to a platform of ex vivo gene therapies developed for the treatment of sickle cell disease (SCD) and β-thalassemia. This platform utilizes self-inactivating lentiviral vectors (such as the BB305 vector or the bifunctional βAS3m/miR7m vector) to transduce autologous CD34+ hematopoietic stem and progenitor cells (HSPCs) ex vivo. The vector delivers a functional, modified human β-globin gene (typically encoding the anti-sickling βA-T87Q-globin variant or βAS3-globin) under the control of a β-globin promoter and locus control region. Following myeloablative conditioning (usually with busulfan), the gene-modified HSPCs are reinfused into the patient. These cells engraft in the bone marrow and differentiate into red blood cells that produce therapeutic anti-sickling hemoglobin, which inhibits the polymerization of abnormal sickle hemoglobin (HbS), thereby preventing red blood cell sickling, hemolysis, and vaso-occlusive crises.

Other names

anti-sickling beta-globin lentiviral vectoranti-sickling β-globin lentiviral vectorlentiviral vector encoding anti-sickling beta-globinlentiviral vector encoding anti-sickling β-globin