Novel lentiviral gene therapy vector improves hemoglobin production in blood disorders

Researchers from the Children’s Hospital of Philadelphia (CHOP; PA, USA) have developed a gene therapy lentiviral vector that can achieve significantly more hemoglobin production than those currently used in trials for the treatment of blood disorders such as sickle cell disease and beta-thalassemia. Their research was recently published in Molecular Therapy. "These results have many potential benefits for the successful treatment of patients affected by beta-globinopathies like sickle cell disease and beta-thalassemia, including a better dose response, a minimized chance of clonal expansion and tumorigenesis, a reduced cost of therapy, and a potentially reduced need for chemotherapy or radiation before...