Gene Therapy Could Save Kids From a Lifetime of Eating Cornstarch

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), can't 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