Duke Researchers Call Gene Therapy A Promising Strategy For Sickle Cell Anemia

efective protein and creates new working copies of the globin protein. This corrected gene is also designed to make a fetal form of the globin protein. The fetal globin protein is normally made before birth and is shut down when mature globin begins being made after birth. But in some people, small amounts of fetal globin continue to be produced. In sickle cell patients who make some fetal globin, the disease symptoms are greatly reduced because fetal globin not only carries oxygen effectively, but also inhibits the mutant globin protein from sickling.

In their experiments, the Duke Medical Center scientists collected blood from sickle cell patients and from umbilical cord blood, the afterbirth of normal infants. From this blood they isolated precursor red blood cells -- the cells that produce mature red blood cells. They added ribozyme molecules that carried the corrected fetal globin genetic sequence into the cells using slippery, fatty spheres called liposomes. Once inside the cell, the ribozymes located the faulty RNA by matching up letters of the genetic code to the defective globin RNA. They then snipped off the defective piece and added in the corrected sequence.

When the scientists broke apart the cells and read the genetic sequence of the globin RNA, in each case the ribozyme had spliced in the new sequence correctly.

Next, the researchers will test the procedure in an animal model of sickle cell disease to see if the corrected globin gene can prevent development of sickling. If this procedure works, Sullenger and his colleagues would like to begin testing the ribozyme therapy in sickle cell patients within two to three years.

The most likely candidates to test the ribozyme therapy would be sickle cell patients who have developed a rare condition called alloimmunization. Patients with severe sickle cell disease often require blood transfusions to replenish their supply of healthy red blood cells. Some of these

Contact: Karyn Hede George
Duke University Medical Center

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