Gene-delivering viruses reach the brain in step toward gene therapy for neurological diseases

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Gene therapies can treat, even potentially cure, certain genetic diseases, but it is challenging to deliver the treatments to the parts of the body where they are needed. Researchers have engineered viruses called adeno-associated viruses (AAVs) to deliver cargo—such as a functioning copy of a gene—to specific cells and organs, but they don’t always get to their desired destination.

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