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UF makes gene therapy advance in severe genetic disorder
Date:5/28/2009

GAINESVILLE A dog born with a deadly disease that prevents the body from using stored sugar has survived 20 months and is still healthy after receiving gene therapy at the University of Florida putting scientists a step closer to finding a cure for the disorder in children.

Called glycogen storage disease type 1A, the genetic disease stops the body from being able to correctly store and use sugar between meals. In order to survive, children and adults with this disease must receive precise doses of cornstarch every few hours. The disease is even more dire in dogs, which must be fed sugar every 30 minutes to survive.

"Without treatment, these dogs all die," said David Weinstein, M.D., M.M.Sc., director of the UF Glycogen Storage Disease Program and co-investigator on the study. "People usually survive because they are fed so much as infants. But by 4 to 6 months of age, they will have developmental delays and a big liver. If it is diagnosed at that point, the kids can do fine. If it is not diagnosed, then the kids get exposed to recurrent low sugars, and they will end up with brain damage, seizures or they will die."

UF researcher Cathryn Mah, Ph.D., a member of the Powell Gene Therapy Center and UF Genetics Institute, will present the findings at an American Society of Gene Therapy meeting this weekend in San Diego.

About one in 100,000 children have this severe form of glycogen storage disease. Children receive doses of cornstarch at scheduled intervals throughout the day because it metabolizes more slowly than other carbohydrates. Until this therapy was discovered about 30 years ago, most children born with this disease did not survive past infancy.

Glycogen storage disease type 1A stems from a faulty enzyme that doesn't convert stored sugar, or glycogen, to glucose, the type of sugar the body uses for energy. This prevents the body from getting the energy it needs and causes glycogen to build up in the liver.'/>"/>

Contact: April Frawley Birdwell
afrawley@ufl.edu
352-273-5817
University of Florida
Source:Eurekalert

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