Intravascular AAV9 administration for delivering RNA silencing constructs to the CNS and periphery

Brett D. Dufour, Jodi L. McBride

    Research output: Chapter in Book/Report/Conference proceedingChapter

    4 Scopus citations


    Viral vector delivery of RNA silencing constructs, when administered into vasculature, typically results in poor central nervous system (CNS) transduction due to the inability of the vector to cross the blood-brain barrier (BBB). However, adeno-associated virus serotype 9 (AAV9) has the ability to cross the BBB and robustly transduce brain parenchyma and peripheral tissues at biologically meaningful levels when injected intravenously. Recent work by our lab has shown that this method can be used to deliver RNA silencing constructs, resulting in signifi cant reductions in gene expression in multiple brain regions and in peripheral tissues. Here, we outline a method for delivery of AAV9 vectors expressing RNA interference (RNAi) constructs that lead to robust simultaneous transduction of mouse peripheral tissues and the CNS following a single injection into the jugular vein. Additionally, we outline methods for necropsy and immunofl uorescence to detect AAV9 transduction patterns in the rodent CNS following a vascular delivery.

    Original languageEnglish (US)
    Title of host publicationMethods in Molecular Biology
    PublisherHumana Press Inc.
    Number of pages15
    StatePublished - 2016

    Publication series

    NameMethods in Molecular Biology
    ISSN (Print)1064-3745


    • AAV9
    • Gene therapy
    • Jugular vein
    • RNAi
    • Systemic
    • Vascular

    ASJC Scopus subject areas

    • Molecular Biology
    • Genetics


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