What made this test unique was that it was used to treat a variety of diseases inside a single animal.

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Seven dogs - all siblings - were used, and each of them had mucopolysaccharidosis VII (MPS VII), also known as Sly syndrome, an enzyme-deficient condition.

MPS VII in humans can indicate Tay Sachs disease and Gaucher disease, both of which are caused by certain enzyme deficiencies.

The MPS VII led to the clouding of the corneas, cardiac disease and bone abnormalities.

Gene therapy slows disease
The study showed that "newborn dogs treated with hepatic gene therapy maintained near-normal mobility throughout the 17-month study and showed little evidence of the other debilitating signs normally associated with the disease," according to a statement from the University of Pennsylvania School of Veterinary Medicine and Washington University of St Louis.

The results will be reported this week on the Web site of the Proceedings of the National Academy of Sciences.

Most patients die in childhood from MPS diseases. The condition is found in one-in-27 000 births among humans, experts say. Symptoms include growth retardation, mobility problems, facial deformities, corneal clouding, heart-valve and liver abnormalities and mental retardation.

The researchers, led by Mark Haskins of the University of Pennsylvania School of Veterinary Medicine and Katherine Parker Ponder of Washington University School of Medicine in St. Louis, also say they hope that this type of gene therapy may one day be used to treat haemophilia, a debilitating blood disease. – (HealthScout)

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