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Article

Systemic administration of novel engineered AAV capsids facilitates enhanced transgene expression in the macaque CNS

22 Nov 2022

Abstract excerpt

BACKGROUND: Adeno-associated virus (AAV) vectors are a promising vehicle for noninvasive gene delivery to the central nervous system via intravenous infusion. However, naturally occurring serotypes have a limited ability to transduce the brain, and translating engineered capsids from mice to nonhuman primates has proved challenging. METHODS: In this study, we use an mRNA-based directed-evolution strategy in...

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PMID: 36417917
DOI: 10.1016/j.medj.2022.11.002

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