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US Researchers Cure Inherited Liver Disease in Rats With Genetherapy

Posted on: Wednesday, 9 March 2005, 09:00 CST

US researchers cure inherited liver disease in rats with gene therapy

LOS ANGELES, March 7 (Xinhua)-- A single dose of gene-virus combination cured rats of an inherited liver disease, US researchers reported on Monday.

Crigler-Najjar syndrome, in which lack of a gene causes the accumulation of bilirubin, can result in jaundice and brain damage if untreated.

In a paper published in the latest issue of the Proceedings of the National Academy of Sciences, the researchers at Baylor College of Medicine said gene therapy may treat the rare disease easily and safely in the future.

The Crigler-Najjar syndrome is currently treated by placing the person under special UV lights, which is unwieldy and time- consuming.

This disease involves a lack of a gene that is found in the part of the cell where detoxification occurs. In the latest research, scientists used a specially developed adenovirus to carry the gene into the animal's cells.

"This is the first time this disease has been completely cured in long term with a single injection in an adult animal," said Brendan Lee, an associate professor at Baylor College of Medicine who led the study.

The viral vector, as it is called, was manipulated so that it minimized toxic side effects.

The study also points the way to using gene therapy to treat a host of other disease that occur because of the lack of genes in this area of the cell, according to Lee.

The viral vector itself is important because it has no long term effect. It does not become part of the genetic machinery of the cell and poses no risk of causing cancer, the researchers said.

"This approach would be applicable to many diseases where you are trying to put something back, targeting the liver," Lee said in a press release.

While the researchers did not think that cure would be possible in all adults with this technique, they did think it poses a real promise for long-term alleviation of the toxic symptoms of these kinds of diseases.

The treatment could be repeated when needed.

"Fifteen years ago, the goal was to get some correction of the problem," he said. "Most effects were transient. Now with these vectors, long-term correction with no long-term toxicity is possible. We need to find a way to avoid short term toxicity."

But the researchers are hesitant to predict when the treatment can be tried in humans. While the viral vector in study is fairly benign, it is necessary to develop a new type minimizing the body' s immune response immediately right after it is injected into the body, Lee said.

Source: Xinhua News Agency - CEIS

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