One shot of gene therapy spreads through brain in animal study

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From the ventral tegmental area (VTA) of the mouse brain the vector carried genes to other areas along neural pathways. Darker circles represent higher numbers of cells with the transported genes. Credit: Childrens Hospital of Philadelphia
From the ventral tegmental area (VTA), of the mouse brain, the vector carried genes to other areas along neural pathways. Darker circles represent higher numbers of cells with the transported genes. Credit: Children's Hospital of Philadelphia

By targeting a site in a mouse brain well connected to other areas, researchers successfully delivered a beneficial gene to the entire brain—after one injection of gene therapy. If these results in animals can be realized in people, researchers may have a potential method for gene therapy to treat a host of rare but devastating congenital human neurological disorders, such as Tay-Sachs disease.


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