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Gene Therapy Corrected Rare Bleeding Disorder: Study
Date:12/6/2010

ents can go on to have more problems with infections, such as graft-versus-host disease, in which the body basically rejects the foreign elements.

"One of the long-lasting complications is the kids couldn't do this, they couldn't do that, they see themselves as different," Conley said. "Transplants are getting better but we need better therapy, there's no question."

In this study, the researchers inserted a healthy gene capable of producing WAS protein into hematopoietic stem cells (the "granddaddy" cells that give rise to different blood cells), then transferred these stem cells back into the patient using a viral vector. A viral vector is a virus that has been modified to deliver foreign genetic material into a cell.

In fact, the experiment was largely successful, with cells now able to produce WAS protein, resulting in increased platelet counts and improvement of some immune-system cells.

"This is a first step that says you can correct the disease but I think most people would look at it and say the risk of leukemia is something, and that, let's see if we can avoid that," said Conley, whose team at St. Jude is working on a therapy involving a different type of vector. "It's a good start, but I think we have better things coming down the road."

In other news from the conference, another group of German researchers have determined that people who donate peripheral blood stem cells or bone marrow to help save a life don't face any heightened risk of cancer.

Previously there had been some concern that drugs needed to get the stem cells out of the bone marrow and into the bloodstream where they could be accessed might pose a risk of leukemia.

The study was based on questionnaires returned from more than 12,500 donors, which also showed the donors tended to be in good health and were willing to donate again.

Another study found that the drug rituximab (Rituxan), used to treat rheumatoid arthritis and forms of leuk
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