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Article

Capsid antigen presentation flags human hepatocytes for destruction after transduction by adeno-associated viral vectors

13 May 2009

Abstract excerpt

Adeno-associated virus (AAV) vectors are effective gene delivery vehicles mediating long-lasting transgene expression. Data from a clinical trial of AAV2-mediated hepatic transfer of the Factor IX gene (F9) into hemophilia B subjects suggests that CTL responses against AAV capsid can eliminate transduced hepatocytes and prevent long-term F9 expression. However, the capacity of hepatocytes to present AAV...

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PMID: 19436115
DOI: 10.1172/jci36891

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