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Genetic therapy reverses nervous system damage in animal model of inherited human disease

By injecting a therapeutic gene directly into the brain, researchers have treated a naturally occurring genetic disease in cats. This is the first genetic disease affecting the central nervous system to be successfully treated in an animal larger than mice and rats. If this approach can be successfully applied to humans, say the researchers, it might one day treat an entire class of diseases called lysosomal storage disorders which cause severe, sometimes fatal, disabilities in about one in 5,000 births. The members of the research team from the University of Pennsylvania School of Veterinary Medicine, the Children's Hospital of Philadelphia and the U.S. Naval Academy published their findings in this month's issue of the Annals of Neurology

The animals involved in the study are born with a genetic disorder directly analogous to alpha-mannosidosis or AMD, an inherited disease in humans that causes severe mental retardation and skeletal abnormalities. Cats with AMD do not live more than six months. Children born with the worst form of the disease rarely survive into their teens.

"Through gene therapy, we replace a 'broken' gene responsible for alpha-mannosidase with the correct, functioning copy, to dramatic results," said John H. Wolfe, a professor of pathology and medical genetics at the Penn School of Veterinary Medicine and a neurology researcher at Children's Hospital. "The treated cats were markedly improved compared to diseased cats, with better balance and muscle control and fewer tremors."

Although the disease itself is rare, AMD is one of about 50 lysosomal storage diseases, a class of diseases that accounts for a significant portion of the instances of mental retardation in children. Other examples include Tay-Sachs disease, Hunter disease and Pompe disease. In a lysosomal storage disease, cellular debris accumulates within storage areas of cells called lysosomes. In the case of AMD, children are born with a faulty version of the gene
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Source:University of Pennsylvania


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