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Journal of Virology, October 2002, p. 10497-10502, Vol. 76, No. 20
0022-538X/02/$04.00+0     DOI: 10.1128/JVI.76.20.10497-10502.2002
Copyright © 2002, American Society for Microbiology. All Rights Reserved.

Improved Hepatic Gene Transfer by Using an Adeno-Associated Virus Serotype 5 Vector

Federico Mingozzi,1 Jörg Schüttrumpf,1 Valder R. Arruda,1 Yuhong Liu,2 Yi-Lin Liu,1 Katherine A. High,1 Weidong Xiao,1 and Roland W. Herzog1*

Department of Pediatrics, University of Pennsylvania Medical Center, and The Children's Hospital of Philadelphia, Philadelphia, Pennsylvania 19104,1 CNS Gene Therapy Center, Thomas Jefferson University, Philadelphia, Pennsylvania 191052

Received 1 May 2002/ Accepted 3 July 2002

Adeno-associated viral (AAV) vectors have been shown to direct stable gene transfer and expression in hepatocytes, which makes them attractive tools for treatment of inherited disorders such as hemophilia B. While substantial levels of coagulation factor IX (F.IX) have been achieved using AAV serotype 2 vectors, use of a serotype 5 vector further improves transduction efficiency and levels of F.IX transgene expression by 3- to 10-fold. In addition, the AAV-5 vector transduces a higher proportion of hepatocytes (~15%). The subpopulations of hepatocytes transduced with either vector widely overlap, with the AAV-5 vector transducing additional hepatocytes and showing a wider area of transgene expression throughout the liver parenchyma.

* Corresponding author. Mailing address: The Children's Hospital of Philadelphia, Abramson Research Center, Rm. 310, 34th St. and Civic Center Blvd., Philadelphia, PA 19104. Phone: (215) 590-4907. Fax: (215) 590-3660. E-mail: rwherzog{at}mail.med.upenn.edu.

Journal of Virology, October 2002, p. 10497-10502, Vol. 76, No. 20
0022-538X/02/$04.00+0     DOI: 10.1128/JVI.76.20.10497-10502.2002
Copyright © 2002, American Society for Microbiology. All Rights Reserved.



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