The Pharmaceutical Journal
Vol 274 No 7340 p292
12 March 2005

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Single injection cures liver disease

Rats with an inherited liver disease have been successfully treated with a single injection of a gene carried by an adenovirus.

US scientists used a manipulated adenovirus to to administer gene therapy to a rat model of Crigler-Najjar syndrome, a liver disease characterised by hyperbilirubinemia.

After one injection, plasma bilirubin levels in the rats were seen to reduce. After less than two years analysis of the bile showed the rats to be free of the disease.

The scientists say this was achieved with negligible chronic toxicity. The study was published early online in the Proceedings of the National Academy of Sciences.