Gene Corrected Removed Bleeding Disorder observed therapy, Study (HealthDay)
SUNDAY, December 5 HealthDay News)--June therapy use, researchers report that German géré '' correct '' a young for Wiskott-Aldrich Syndrome, a rare but disability katastwofik children leads to bleeding extension of hits you have minor scrapes, and let these children susceptible to certain cancers and dangerous infection.
However, one in 10 children in the study developed acute T-cell leukemia, apparently as a result of living vecteur used to young healthy: the boy is currently on chemotherapy, study author.
'' This is a very interesting isn't the first, but a little afraid and we need to move to plus vecteurs, '' said Dr. Mary Ellen Conley, program director genetic Immunodeficiencies in hospital St. Jude children's research in Memphis, Tenn.
"The study shows evidence-two-a set of rules, which is young and stem cells therapy in a genetic disability like that great, '' added Paul Sanberg, a specialist stem cell Director for the University of South Florida Center Path for aging and repair brain in Tampa.
Or Conley Sanberg or involved in the study, which is scheduled to present on Sunday at the annual meeting of the American Society of tid blood in Orlando, Fla.
According to Conley, the son (mostly men) and Wiskott-Aldrich Syndrome (WAS) with a genetic hérité eyes on chromosome X goes number and size plaquettes and iron remarquablement sensitive to facile are bleeding and infection, including different types of cancer.
Greffes bone marrow is the main treatment for the disorder, if they succeed, well basically those who are sick.
"Growth, in college and they cause any trouble," Conley said. '' But they will take it easy a group of patients beginning to get better. "
Even if a good game, transplant recipients can have more problems with infection, such as graft-par-host disease, where the body basically rejected foreign elements.
"One of the side effects are long-durable children couldn't do that, it sees itself as different," Conley said. "Greffes that began the best but we need therapy better, there is no question."
In this study, join a young healthy protein WAS able to produce in stem cell hématopoïétique red globil ("granddaddy '' in it give rise to different cell), when transferred package these stem cells into the patient using a vecteur alive. A living vecteur is a virus that was modified to deliver foreign genetic material in a cell.
In fact, the experience was largely successful, and now produces protein, WAS raised platelet counts and improvement package red blood cells istem defense/some red blood cells.
'' This is a first step you have to correct the disease but I think most people would see, said the risk of leukemia is something, and, let's see if we can avoid '' Conley, in St. Jude team is working on a therapy involving a different type of vecteur said. '' This is a great start, but I think we have the best down the road. "
In other news conference, another German teams of researchers determined that people see on blood stem cells or bone marrow for the life of one not facing overall cancer accrue.
Précédemment some anxiety that drugs needed for stem cells out of his land's bone marrow and in the bloodstream where they could make accessible represent a risk of leukemia.
The study was based on questionnaires returned to donors more than 12,500, which also showed that donors in good health and not willing to give.
Another study found the drug rituximab (Rituxan), used to treat pain in joints and form leukemia and UN, many could reduce graft-par-host disease of stem cell transplant recipients.
More information
National marrow donor program will be more about Wiskott-Aldrich Syndrome.
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