A single dose of gene-virus combination cured rats of a inherited liver disease in which lack of a gene causes the accumulation of bilirubin –which, untreated, results in jaundice and brain damage, said researchers at Baylor College of Medicine in a report in the Proceedings of the Natural Academy of Sciences.
"This is the first time this disease (Crigler-Najjar syndrome) has been completely cured long term with a single injection in an adult animal," said Dr. Brendan Lee, associate professor of molecular and human genetics and a Howard Hughes Medical Investigator at Baylor College of Medicine. Crigler-Najjar syndrome is currently treated by placing the person under special UV lights. It is an unwieldy and time-consuming treatment.
While Lee does not think that cure would be possible in all adults with this technique, he does think it poses a real promise for long-term alleviations of the toxic symptoms of these kinds of diseases. The treatment could be repeated when needed.
Ross Tomlin | EurekAlert!
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