Abstract
Recent clinical trial data of adeno-associated virus (AAV) 2-mediated hepatic gene transfer of factor IX into hemophilia B subjects demonstrated AAV2-specific CD8+ T cells and identified an immunodominant AAV2 epitope. Expansion of this CD8+ T cell population coincided with an asymptomatic rise of liver transaminases, and correlated with subsequent loss of transgene expression. This suggests that CD8+ T cells directed against AAV capsid antigen clear the transduced hepatocytes. Others in our laboratory have directly shown the lysis of AAV-transduced hepatocytes by CD8+ T cells in vitro.
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