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  1. No Access

    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

    Epigenetic Downregulation of Scn3a Expression by Valproate: a Possible Role in Its Anticonvulsant Activity

    Upregulation of sodium channel SCN3A expression in epileptic tissues is known to contribute to enhancing neuronal excitability and the development of epilepsy. Therefore, certain strategies to reduce SCN3A expres...

    Na-Na Tan, Hui-Ling Tang, Guo-Wang Lin, Yong-Hong Chen, ** Lu… in Molecular Neurobiology (2017)

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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)