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Gene therapy 'can reverse muscular dystrophy'
Lab worker
Gene therapy can treat previously incurable disease
Scientists have used gene therapy to reverse the crippling effects of muscular dystrophy in animals.

A team from the Children's National Medical Center in Washington and the University of Pittsburgh has become the first to show that whole muscles can be restored to full fitness using gene therapy.

They presented their findings to the annual meeting of the American Society of Human Genetics in San Francisco on Friday.

Researcher Devin Dressman said: "We are very excited by these preliminary results, which suggest that we can use a non-toxic virus to safely shuttle a gene for an important muscle protein that is improperly made in people suffering limb girdle muscular dystrophy.

"These findings demonstrate the feasibility of using the gene therapy approach in treating affected patients."

In their experiments, the investigators used a non-replicating adeno-associated virus (AAV) to carry a gene that stimulates production of the sarcoglycan protein, an important constituent of skeletal muscle.

AAV is ideal for the task because it does not provoke an immune response from the body.

Injected into hamster

The AAV-sarcoglycan gene combination was injected into the leg muscles of a hamster with limb girdle muscular dystrophy.

One month later the muscles had increased by nearly 100% in strength and had resumed normal size.

Limb girdle muscular dystrophy causes rapid degeneration of the large muscles attached to the shoulders and hips.

There is no current cure, and the disease is fatal.

The researchers hope that their technique can be modified to treat other common forms of muscular dystrophy, such as the lethal condition, Duchenne muscular dystrophy.

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