AAV-PHP.B Administration Results in a Differential Pattern of CNS Biodistribution in Non-human Primates Compared with Mice

William A. Liguore, Jacqueline S. Domire, Dana Button, Yun Wang, Brett D. Dufour, Sathya Srinivasan, Jodi L. McBride

    Research output: Contribution to journalArticle

    4 Scopus citations

    Abstract

    Liguore and colleagues characterize biodistribution of AAV-PHP.B in the rodent and rhesus macaque CNS following intra-CSF and intravascular delivery modalities. Although equal transduction efficiency was seen in mice regardless of delivery route, intra-cisterna magna infusion led to superior transduction efficiency compared with intra-carotid artery infusion in the rhesus macaque CNS.

    Original languageEnglish (US)
    Pages (from-to)2018-2037
    Number of pages20
    JournalMolecular Therapy
    Volume27
    Issue number11
    DOIs
    StatePublished - Nov 6 2019

    Keywords

    • AAV-PHP.B
    • AAV9
    • CNS
    • adeno-associated virus
    • brain
    • gene therapy
    • gene transfer
    • mouse
    • rhesus macaque
    • viral transduction

    ASJC Scopus subject areas

    • Molecular Medicine
    • Molecular Biology
    • Genetics
    • Pharmacology
    • Drug Discovery

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