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Development of AAV Variants with Human Hepatocyte Tropism and Neutralizing Antibody Escape Capacity

3 Jun 2020

Abstract excerpt

Adeno-associated virus (AAV) vectors have been successfully used in patients with bleeding disorders and blindness. For human liver targeting, two major factors restrict effective AAV transduction after systemic administration of AAV vectors: human hepatocyte tropism and neutralizing antibodies (Nabs). In this study, we attempted to isolate AAV variants with the ability to transduce human hepatocytes and escape...

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PMID: 32637455
DOI: 10.1016/j.omtm.2020.06.003

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