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Gene therapy could save kids from a lifetime of eating cornstarch
Date:3/12/2008

DURHAM, N.C. A gene therapy treatment that restores a missing liver enzyme in test animals could provide a cure for a rare metabolic disorder in humans, according to Duke University Medical Center researchers.

People born with the disorder, called glycogen storage disease type Ia (GSD-Ia), cant make an enzyme that helps the liver store and release glucose, the sugar that all cells use for energy. Without treatment, their blood sugar levels drop dangerously low, causing seizures and organ damage. Eating raw cornstarch, a slowly digested carbohydrate, and avoiding dietary sugar can help people with GSD-Ia maintain their glucose levels. However, even a special diet does not prevent the eventual liver damage that results from the absent enzyme, and many adults with the disease develop liver and kidney failure or liver cancer. With treatment, most people with GSD-1a have a relatively normal lifespan

The gene therapy developed at Duke would give liver cells the correct genetic code for manufacturing the enzyme. A modified virus transfers the enzyme genes by infecting liver cells. The virus is not linked to any known human disease, and cannot copy itself and spread to other people, said medical geneticist Dwight Koeberl, M.D., Ph.D., lead study author and an associate professor in the Department of Pediatrics.

The research involved creating a virus so focused on targeting liver cells that only a tiny amount is needed for treatment, minimizing potential side effects. Showing that the virus is safe and effective in small doses is an important step in bringing the treatment to clinical trials in humans.

The gene therapy replaced the missing enzyme in the liver to fully normal levels, and protected both mice and dogs with the disease from low blood glucose for up to a year. No one has fully corrected the enzyme that produces glucose in the liver before. We think we can correct every cell in the liver, Koeberl said.

The results
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Contact: Debbe Geiger
Debbe.Geiger@duke.edu
919-660-9461
Duke University Medical Center
Source:Eurekalert

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