Adeno-associated viral vector-mediated gene transfer of human blood coagulation factor IX into mouse liver.

  title={Adeno-associated viral vector-mediated gene transfer of human blood coagulation factor IX into mouse liver.},
  author={Hiroyuki Nakai and Roland W. Herzog and J. Nathan Hagstrom and Jurgen Walter and Sheng H. Kung and Eun Young Yang and Shing Jen Tai and Yoko Iwaki and Gary J. Kurtzman and Krishna J. Fisher and Peter Colosi and Linda B. Couto and K. A. High},
  volume={91 12},
Recombinant adeno-associated virus vectors (AAV) were prepared in high titer (10(12) to 10(13) particles/mL) for the expression of human factor IX after in vivo transduction of murine hepatocytes. Injection of AAV-CMV-F.IX (expression from the human cytomegalovirus IE enhancer/promoter) into the portal vein of adult mice resulted in no detectable human factor IX in plasma, but in mice injected intravenously as newborns with the same vector, expression was initially 55 to 110 ng/mL. The… CONTINUE READING
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