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Last Updated:  Monday, 24 February, 2003, 00:23 GMT
Gene therapy breakthrough
Ultrasound image
The technique uses tiny bubbles
Scientists have developed a more effective way to carry out gene therapy without the risks of current methods.

The modification of genes offers the potential to treat or cure many diseases.

But the use of viruses to deliver the modified genes to the target tissue is not always safe, and other methods do not seem to work well.

The latest technique, developed by scientists at Imperial College London and the Medical Research Council in the UK, appears to be effective - and does not require the use of viruses.

It works by combining the use of microbubbles and ultrasound.

So far it has been tested on skeletal muscle in mice, but it is hoped it may eventually be used to treat muscular dystrophy in children.

Tiny bubbles

We need to build on the research to improve the technique and assess the possible impact it could have on diseases such as muscular dystrophy, cancer, and diseases of the heart
Dr Martin Blomley
Microbubbles are already in use to improve patient ultrasound scans in the heart, liver and many other areas and are known to be both safe and effective.

They are tiny gas bubbles measuring about three microns, and are usually injected intravenously to boost ultrasound signals.

But there is also evidence that when ultrasound is applied to microbubbles, the microbubbles pop and this can cause small perforations in the target cells which allow the DNA to enter.

This enabled the researchers to develop a "point and shoot" approach to gene therapy.

They mixed a type of microbubble already in use by doctors for scanning patients with modified DNA and injected it into the skeletal muscle cells of mice of different ages.

The target cells were then subjected to ultrasound to disrupt them, and make them more likely to take up the modified DNA.

The technique proved to be 10 times more effective than more conventional methods.

Even the use of microbubbles alone without ultrasound proved to be more effective, particularly in younger mice.

In addition, the amount of inflammation and damage associated with the injection was reduced when microbubbles were used.

Great promise

Currently, most gene therapy uses viruses to transport the modified genes to their intended location.

However, this can lead to infection of non-target tissues, and can stimulate dangerous immune responses.

Researcher Dr Martin Blomley said: "What we've found here seems a promising lead into a new, safe and effective way of delivering genes into target cells.

"Gene therapy holds great promise in future for curing and ultimately preventing serious diseases but is still in its infancy as a clinical tool.

"This promising study suggests that there may be a less invasive and more efficient, safe and accurate technique for targeting tissue, than those currently in use."

"Now we've found a good delivery system, we need to build on the research to improve the technique and assess the possible impact it could have on diseases such as muscular dystrophy, cancer, and diseases of the heart."

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