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The Neurotropic Properties of AAV-PHP.B Are Limited to C57BL/6J Mice

Overview
Journal Mol Ther
Publisher Cell Press
Specialties Molecular Biology
Pharmacology
Date 2018 Feb 12
PMID 29428298
Citations 224
Authors
Juliette Hordeaux
Qiang Wang
Nathan Katz
Elizabeth L Buza
Peter Bell
James M Wilson
Affiliations
Soon will be listed here.
Abstract

Improved delivery of adeno-associated virus (AAV) vectors to the CNS will greatly enhance their clinical utility. Selection of AAV9 variants in a mouse model led to the isolation of a capsid called PHP.B, which resulted in remarkable transduction of the CNS following intravenous infusion. However, we now show here that this enhanced CNS tropism is restricted to the model in which it was selected, i.e., a Cre transgenic mouse in a C57BL/6J background, and was not found in nonhuman primates or the other commonly used mouse strain BALB/cJ. We also report the potential for serious acute toxicity in NHP after systemic administration of high dose of AAV.

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