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In vivo AAV-fVIII gene therapy. AAV-fVIII vectors selected for hepatocyte tropism and encompassing a fVIII transgene cassette under a liver-specific transcriptional promoter are infused into adult patients via peripheral vein. Once in circulation, the AAV vectors are thought to transduce primarily hepatocytes, persist episomally, and direct biosynthesis and secretion of fVIII into the bloodstream.
