Gene Therapy Reverses Muscular Dystrophy

Using a novel approach, scientists at the Children's National Medical Center and the University of Pittsburgh have successfully repaired muscles in hamsters that were ravaged by a form of muscular dystrophy called limb girdle muscular dystrophy.

In the study, the researchers used a non-replicating adeno-associated virus (AAV) as a gene vector. The virus carried a gene for a component of skeletal muscle called sarcoglycan protein. This protein is not made properly by people suffering from limb girdle muscular dystrophy. AAV is unique in that it does not provoke a response from the immune system and can reside in cells without initiating a response. Once integrated within a cell, it will stay there permanently. This allows the sarcoglycan protein to be produced over the long term.

The AAV vector along with the sarcoglycan gene was injected into the leg muscles of hamsters with limb girdle muscular dystrophy. After approximately a month, the treated muscles were tested and the results were startling. The muscles had increased almost 100 percent in strength and were nearly normal in size. This was the first time that researchers had observed entire muscles being functionally repaired and restored after being ravaged by the effects of muscular dystrophy.

Limb girdle muscular dystrophy causes degeneration of the large muscles attached to the hips and shoulders. It is fatal and has no known effective treatment. A more common variant called Duchenne muscular dystrophy affects some 20,000 individuals in the United States alone.

The researchers are currently exploring ways to develop a treatment protocol based on their findings in this study. They believe that using the non-toxic gene vector will make it possible to safely and effectively "shuttle" the gene to muscles affected by limb girdle muscular dystrophy and perhaps other forms of muscular dystrophy as well.

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