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

By Amanda Gardner
HealthDay Reporter

SUNDAY, Dec. 5 (HealthDay News) -- Using gene therapy, German researchers report that they managed to "correct" a malfunctioning gene responsible for Wiskott-Aldrich syndrome, a rare but devastating childhood disorder that leads to prolonged bleeding from even minor hits or scrapes, and also leaves these children vulnerable to certain cancers and dangerous infections.

However, one of the 10 kids in the study developed acute T-cell leukemia, apparently as a result of the viral vector that was used to insert the healthy gene. The boy is currently on chemotherapy, the study authors noted.

"This is a very good first step, but it's a little scary and we need to move to safer vectors," said Dr. Mary Ellen Conley, director of the Program in Genetic Immunodeficiencies at St. Jude Children's Research Hospital in Memphis, Tenn.

"The study shows proof-of-principle that gene therapy with stem cells in a genetic disorder like this has strong potential," added Paul Sanberg, a stem cell specialist who is director of the University of South Florida Center of Excellence for Aging and Brain Repair in Tampa.

Neither Conley nor Sanberg were involved in the study, which is scheduled to be presented Sunday at the annual meeting of the American Society of Hematology in Orlando, Fla.

According to Conley, children (mostly boys) with Wiskott-Aldrich syndrome (WAS) are born with an inherited genetic defect on the X chromosome that affects the number and size of platelets and makes the children remarkably susceptible to easy bleeding and infections, including different types of cancer.

Bone marrow transplants are the main treatment for the disorder which, if they succeed, basically cure the patient.

"They grow up, go to college and they cause problems," said Conley. "But they're not an easy group of patients to transplant."

Even if a good match is found, transplant recipi
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