Word: thalassemia
(lookup in dictionary)
(lookup stats)
Dates: all
Sort By: most recent first
(reverse)
...first candidates for this therapy would be people with life-threatening hereditary disorders that are caused by a single, known defective gene. Among the illnesses being considered for gene therapy: beta-thalassemia, a severe form of anemia, and three rare disorders caused in each case by a defect in a gene that orders the production of a single, vital protein...
...devised by scientists at the National Heart, Lung and Blood Institute in Bethesda, Md., the University of Illinois College of Medicine in Chicago and Johns Hopkins University in Baltimore, is based on experiments on baboons. It has been used in only a handful of human patients suffering from severe thalassemia or sickle cell anemia. These blood disorders result from defects in the genes that control production of hemoglobin, the substance that carries oxygen in the blood. In essence, thalassemia victims cannot form healthy red blood cells on their own and require periodic transfusions; sickle cell patients are subjected to painful...
...drug involved is 5-azacytidine, which until now has been used only to treat cancer. In the most fully described case so far, a 42-year-old man with severe thalassemia, who needed a blood transfusion every two weeks, received a continuous infusion of the drug through a vein in his arm for seven days. At the end of the week, the concentration of healthy red blood cells in his blood had increased by 25%. This beneficial effect persisted for about a month...
Scientists have learned in recent years that several specific genetic defects occurring in human cells cause beta thalassemia, an incurable blood disorder that can lead to anemia, bone deformation and early death. The problem has been to find a way to reprogram the errant genetic messages. Now a series of experiments, directed by Dr. Yuet Wai Kan of the University of California at San Francisco, has brought researchers a step closer to a cure for one type of the disease...
...team first constructed an artificial gene that could replace the instructions of a faulty gene subunit that was causing beta thalassemia. When frog egg cells were then injected with both the man-made instructor and the defective genetic material, the fault was corrected. The successful experiment, published in the British journal Nature, marks the first reported time an artificially constructed gene has been successfully used to correct a human genetic defect...