The Reporter: December 1997, Vol.8, No.5
Research notes
Gene Therapy for Sickle Cell Disease

 The long-term transfer and highlevel long-term expression of the normal human beta globin gene in an animal model has been demonstrated for the first time by P&S researchers. The study may lead to gene therapy for the treatment of sickle cell disease and beta thalassemia, a related disorder. Senior author Dr. Arthur Bank, P&S professor of medicine and genetics and development, and colleagues put a human beta globin gene into a safe retrovirus and added the virus to mice bone marrow cells in vivo. The modified cells were then transplanted into mice. The researchers were able to detect the presence of the human beta globin gene up to eight months later.

 The researchers documented high levels of expression of the gene. In one mouse, 20 percent of the total beta globin it produced was from the human beta globin gene."If we could attain that level of normal human beta globin gene expression in human marrow cells, it would be enough to expect to ameliorate, if not cure, the anemia of patients with sickle cell disease and beta thalassemia," says Dr. Bank.

 The researchers are investigating improved gene transfer systems in mouse models of sickle cell disease and beta thalassemia and developing better ways to transfer retroviruses into human hematopoietic stem cells.