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Article

Superior In vivo Transduction of Human Hepatocytes Using Engineered AAV3 Capsid

29 Mar 2016

Abstract excerpt

Adeno-associated viral (AAV) vectors are currently being tested in multiple clinical trials for liver-directed gene transfer to treat the bleeding disorders hemophilia A and B and metabolic disorders. The optimal viral capsid for transduction of human hepatocytes has been under active investigation, but results across various models are inconsistent. We tested in vivo transduction in "humanized" mice. Methods to...

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PMID: 27019999
DOI: 10.1038/mt.2016.61

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