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    Article

    Toxicity after AAV delivery of RNAi expression constructs into nonhuman primate brain

    RNA interference (RNAi) for spinocerebellar ataxia type 1 can prevent and reverse behavioral deficits and neuropathological readouts in mouse models, with safety and benefit lasting over many months. The RNAi ...

    Megan S. Keiser, Paul T. Ranum, Carolyn M. Yrigollen, Ellie M. Carrell… in Nature Medicine (2021)

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    Article

    Molecular signatures of disease brain endothelia provide new sites for CNS-directed enzyme therapy

    The brain vasculature forms an immense network such that most neural cells are in contact with a microvessel. Here we tested the hypothesis that endothelia lining these vessels can be harnessed to create a cel...

    Yong Hong Chen, Michael Chang, Beverly L Davidson in Nature Medicine (2009)