Gene TransferWhile stem cell transplantation has a high success rate among young, HLA-matched patients, researchers hope to find a more universally available cure. Gene transfer may be the solution. "The goal is to introduce a gene to restore normal function in red blood cells, removing the transfusion requirement," said Michel Sadelain, a researcher at Memorial Sloan-Kettering Cancer Center."The goal is to introduce a gene to restore normal function in red blood cells."The process involves removing some of the patient's stem cells, inserting a gene that codes for the healthy globins, and then re-injecting the cells into the body. To introduce the new gene, Sadelain and his team have created a vector based on a lentivirus that is capable of introducing β-hemoglobin genes into the cells. They demonstrated the concept could work in thalassemic mice in 2003. "These mice remained alive only due to transplanted gene," said Sadelain. In the laboratory, they've shown that the gene vector works at least as well in human cells as it does in mouse cells, yielding a level of expression of about 55%.A clinical trial of gene therapy to cure thalassemia is planned to begin in the next year in France, reported Arthur Bank, a physician and geneticist at Columbia University in New York and Philippe Leboulch at Harvard Medical School and Brigham & Women's Hospital in Boston. The first phase will enroll five patients with β thalassemia and look primarily at safety.