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


Chen solved the problem by using the body's own system for enzyme delivery. He reasoned that the liver secretes a special form of the GAA enzyme that has a molecular signal attached. This special enzyme is recognized by muscle cell receptors, which then trigger the cell to take up the enzyme and direct it to where it is needed within the muscles.

When the researchers injected the virus containing the specially designed genetic information into a mouse that develops Pompe disease, the virus went to the liver, which then began making and secreting the special enzyme into the blood stream of the animals. The researchers hoped that in this manner, the muscle cells could receive the enzyme without having to each be individually injected with the virus. The idea worked -- the mice that received the modified GAA gene in their livers subsequently had reduced accumulation of glycogen in muscles throughout the body.

"The heart and diaphragm muscles appeared to be especially responsive to the treatment," said Amalfitano. "This is significant because failure of the heart or respiratory muscles are the primary cause of death in many people with Pompe disease."

If future studies prove successful, a gene therapy strategy such as the one devised by the researchers could allow the body to generate its own continuous supply of enzyme, by using the liver as an "enzyme factory" and eliminating the need for lifetime injections of the enzyme. "This is the first example of the simultaneous correction of multiple muscle groups after a single, simple, intra-venous administration of a gene therapy vector" Amalfitano said, "a hurdle that has always made the potential of gene therapy to treat muscle diseases very difficult to envision."

A.J. McVie-Wylie, H. Hu, and T. L. Dawson of Duke and N. Raben and P. Plotz of the National Institute of Arthritis and Musculoskeletal and Skin Diseases also contributed to the work.


Contact: Karyn Hede
Duke University Medical Center

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