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Article

AAV8-mediated Hepatic Gene Transfer in Infant Rhesus Monkeys (Macaca mulatta)

2 Aug 2011

Abstract excerpt

Many genetic metabolic diseases manifest in infancy, therefore, it is important to develop effective treatments that could be implemented at this time. Adeno-associated virus serotype 8 (AAV8) gene transfer has been studied in neonatal mouse, cat, and dog models and shown some efficacy with a single hepatic injection at birth. AAV8-mediated liver gene transfer has also generated sustained therapeutic effects in...

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PMID: 21811248
DOI: 10.1038/mt.2011.151

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