New gene therapy could treat red blood cell disorders

A team of researchers has designed a gene therapy strategy that could treat the red blood cell disorders beta-thalassemia disease and sickle cell anemia. They also developed a test that can be used to predict patient response prior to treatment. "This gene therapy technique has the potential to cure many patients, especially if we prescreen them to predict their response using just a few of their cells in a test tube," the study's lead investigator, Dr. Stefano Rivella, said. This is the first time researchers have been able to correlate the outcome of transferring a healthy beta-globin gene into diseased cells with increased production of normal hemoglobin. This is possible by hooking an ‘ankyrin insulator’ to the beta-globin gene carried by a virus. The virus then can be inserted into bone marrow stem cells taken from patients and introduced back to the patient through a bone marrow transplant, leading to the production of healthy beta-globin protein and hemoglobin.