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 journalArticlepeer-review

26 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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