Gene Therapy Corrected Rare Bleeding Disorder: Study (HealthDay)

SUNDAY, December 5, HealthDay News)--using a gene therapy, German researchers are managed in a "fix" a report on the failure of a gene that is responsible for the Wiskott-Aldrich Syndrome, a rare but devastating childhood disorder, which leads to even very small amounts of hits or scrapes prolonged bleeding, and also to exclude those children of certain cancers and dangerous infections.

However, one study, 10 children developed acute T-cell leukemia viral vector, apparently that was used to Add healthy, on account of a gene. The boy is currently the chemotherapy, research the authors note.

"This is very good, first of all, but is a little bit scary and we have moved to safer and healthier the working environment, vectors," said Dr. Mary Ellen Conley, the program's Genetic Immunodeficiencies St. Jude children's Research hospital, Memphis, Tenn. the Director shall

"The study shows proof of principle, such as gene therapy, genetic are in that regard with strong potential of stem cells," will be added to Paul Sanberg, stem cells, which is the expert in Director, South Florida Center for aging and Brain repair Tampa University.

Rather than Conley Sanberg were involved in the investigation, which are designed to be a Sunday Hematology American Society, at the annual meeting, Orlando, Fla.

According to him, Wiskott-Aldrich Syndrome Conley (WAS) (mostly boys) of children born in an inherited genetic deficiency, which affects the number and size of verihiutaleita and sets the value of the children highly vulnerable to easy bleeding and infection, including different types of cancer on the x chromosome.

Are the most important of human Bone marrow disorder which, if they succeed, basically for the treatment of the patient laattavalun.

"They grow, go to College and is causing problems," Conley said. "But they are easy to transplant patients of the group."

Although good match found, recipients can go to elinpankkien more problems, infections, such as a graft-versus-host disease where the body will reject in principle with foreign elements.

"One of the long-lasting complications is children was unable to do this, they failed to do so, they will see themselves as different," Conley said. "Getting the better of human origin are, but we must better therapy does not exist."

In this part of the healthy gene researchers that can be produced as part of the blood stem cells WAS protein (the "granddaddy" cells that cause different blood cells), then move these stem cells are added back to the viral vector to the patient. Viral vector is a virus that has been modified in order to provide you with information of foreign genetic material in the cell.

The experiment was in fact largely successful cells now may make WAS the increased protein, leukocytes counts and some improvement of the immune system cells.

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

News and from the Conference as a member of another group of German researchers have decided that the people who donate bone marrow or peripheral blood stem-cell saves life does not face the risk of cancer turvavälikohtausten.

In the past, there had been some concern that the drugs necessary ran out of bone marrow and stem cell appears in the bloodstream, where they can use could pose a risk for leukemia.

The research was based on the returned more than 12,500 from donors, which also showed an unsatisfactory, it is a good idea to donors and the questionnaire wanted to donate again.

Another study found that such as rheumatoid arthritis and the leukemia and Lymphoma, drug-rituximab (Rituxan), could significantly reduce Graft-versus-host disease stem cell elinpankkien carries.

For more information about the

National Marrow Donor Program is more Wiskott-Aldrich Syndrome.


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