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Last Updated: Sunday, 6 July, 2003, 23:16 GMT 00:16 UK
Muscular dystrophy drug progress
arm DNA
The treatment is aimed at one mutated gene
Scientists are encouraged by the early success of treatment which may eventually help patients with a form of muscular dystrophy.

Duchenne muscular dystrophy is a wasting disease caused by mutations on a particular gene.

It is the most common muscular dystrophy, affecting one in 3,500 children - most of whom die early in life as a result.

The mutations on the gene stop it producing the chemical needed to protect muscle cells and prevent wasting.

Antisense therapy offers huge potential for the effective treatment of diseases like Duchenne muscular dystrophy
Professor Terrence Partridge, Medical Research Council Clinical Sciences Centre
Some experts believe that it may be possible to alleviate the disease by replacing the gene entirely.

However, a slightly different strategy has paid dividends for researchers at the Medical Research Council's Clinical Sciences Centre

Skipping instruction

Instead of trying to insert an entirely new version of the gene - called the dystrophin gene - which is problematic simply because of its large size, scientists are trying to issue the body instructions to ignore the faulty bits.

While this, if successful, does not completely correct the problem, it does mean that a body chemical is produced that is almost as effective as the normal version.

The technique, called "anti-sense" therapy, might also be easier to get working in a drug than full-blown gene therapy.

Drugs are injected which carry small fragments of genetic code instead of an entire gene.

It is these fragments which direct the machinery of the cell to ignore the mutated sections of the gene.

Muscle improvement

In mice bred with a condition similar to Duchenne muscular dystrophy, the antisense therapy managed to restore production of the dystrophin protein - and improved muscle function.

Professor Terrence Partridge, who led the project, said: "Antisense therapy offers huge potential for the effective treatment of diseases like Duchenne muscular dystrophy.

"However, before this therapy can be tested on humans, more work must be done to improve the current method of delivering the antisense drug."

Other experts are hopeful some form of gene therapy will be able to help those with the wasting disease.

A research team from the University of Washington School of Medicine has managed to find a way to replace the dystrophin gene in mice by "infecting" muscle cells with a modified cold virus.

This is a breakthrough because the sheer size of the gene has made this difficult.

In these experiments too, mice had improved muscle function as a result of the treatment.


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