One-shot gene therapy successful in mice

Philadelphia -- U.S. scientists, in a study that might lead to genetic disease treatments, have discovered one shot of gene therapy can spread through the brain of an animal.

By targeting a site in a mouse brain well connected with other areas, researchers at the Children's Hospital of Philadelphia and the University of Pennsylvania successfully delivered a beneficial gene to the entire brain in one injection of gene therapy.

If the results in animals can be replicated in people, the finding might lead to gene therapy that could treat many congenital human neurological disorders, such as Tay-Sachs disease.

The research appeared in the Journal of Neuroscience.

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