DURHAM, N.C. -- In a first step toward an effective treatment for sickle cell anemia, researchers at Duke University Medical Center have shown that they can use a new type of gene therapy to correct the defect in human blood cells.
The results of their laboratory studies, published in the June 5 issue of the journal Science, show that successful gene therapy may lie not in correcting faulty DNA, the storehouse of genetic information, but in correcting the RNA, which translates that genetic information to the protein synthesis machinery of a cell. It also provides a potential path toward ridding the sickle cell defect from people born with the debilitating disease.
The researchers plan to begin testing the therapy in sickle cell patients within a few years.
"We have shown for the first time that it is possible to correct a genetic defect in blood extracted from patients for experimentation, not just in laboratory-grown cells," said Bruce Sullenger, the paper's senior author. "In a disease like sickle cell anemia, if we could get even 10 to 20 percent correction of the defect, it could make a huge difference for patients."
Sullenger and his colleagues, Ning Lan, Dr. Richard Howrey, Dr. Seong-Wook Lee and Dr. Clayton Smith, all from Duke's Center for Genetic and Cellular Therapies, tried their new therapy on sickle cell anemia because there is no effective treatment for the underlying genetic defect and because the gene is under complex genetic controls that make it unsuitable for other types of gene replacement therapy. The research was funded in part by a grant from the National Heart, Lung and Blood Institute to Sullenger and from a Korean Academic Research Fund grant to Lee.
Sickle cell anemia is an inherited disease that is most common among
people whose ancestors come from Africa and the Middle East. About one in 12
African Americans carries the sickle cell trait. The red blood cells of people
Contact: Karyn Hede George
Duke University Medical Center