Duke scientists reverse a rare form of muscular dystrophy in mice using gene therapy

zyme and replacing the faulty gene. The first method uses cells grown in the laboratory that secrete a special form of GAA that, when injected intravenously, is easily taken up by muscle cells and processed to a useful form. Chen and his colleagues have developed a way to make the enzyme in large quantities and licensed that technology to Synpac (North Carolina) Inc., a drug development company in Research Triangle Park, and its parent company, Synpac Pharmaceuticals Ltd. of Cambois, England. The company is funding an on-going clinical trial to test the enzyme therapy in up to three infants at Duke. The research is also supported by the Howard Hughes Medical Institute and a gift from the Garrette Foundation.

"We are hopeful that the enzyme therapy will be an efficacious first therapy for this devastating disease," Chen said. "However, we ultimately would like to correct the defect at the genetic level and this study shows us that this might be possible."

Chen collaborated with Dr. Andrea Amalfitano, a pediatric geneticist who studies ways to get genes inside cells, and the two designed an experimental system to deliver the genetic information encoding the GAA enzyme using a modified adenovirus, the common cold virus. Amalfitano has developed a form of the virus that appears to be able to evade detection by the immune system more so than other viruses used in gene therapy. The modified virus tends to normally infect liver cells since the liver filters all blood within the body, Amalfitano reported. He and his colleagues published the results of this work in the February 1999 issue of Human Gene Therapy.

"The liver normally makes and secretes a large number of enzymes and we used that to our advantage in designing our gene delivery system," said Amalfitano. "We had to find a way to get the enzyme from the liver to all the muscles in the body, which is the major hurdle to overcome in designing gene treatments for muscle disorders.

Contact: Karyn Hede
Duke University Medical Center

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