Purpose: Stimulating expression of the normal fetal globin genes is a preferred method of
ameliorating sickle cell disease and [beta]-thalassemia for the majority of patients in North
America who do not have appropriate bone marrow donors. Patients and Methods: Due to
increased survival of red blood cells that contain both hemoglobin S and hemoglobin F, as
little as 4-8% fetal globin synthesis in the bone marrow can produce levels of hemoglobin F
of~ 20% in the peripheral circulation. Some success has been achieved in stimulating …