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Semin Liver Dis 1999; 19(1): 61-69
DOI: 10.1055/s-2007-1007098
ORIGINAL ARTICLE

© 1999 by Thieme Medical Publishers, Inc.

Hepatic Gene Therapy Using Adeno-associated Virus Vectors

Gijsbert A. Patijn1 , Mark A. Kay2
  • 1Department of General Surgery, Leiden University Medical Center, Leiden, The Netherlands
  • 2Department of Pediatrics and Genetics, Stanford University School of Medicine, Stanford, California
Further Information

Publication History

Publication Date:
17 March 2008 (online)

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ABSTRACT

Recombinant adeno-associated virus vectors have been shown to safely transduce a number of tissues in preclinical animal studies. The level of gene transfer is sufficient to successfully treat a large number of medical disorders. Moreover, the long-term persistence of the vector sequences in animals makes it likely that this vector will be useful for genetic diseases requiring life-long therapy. This review outlines the biological principles of the vector, as well as its advantages and current limitations as it relates to use in hepatic gene therapy.

KEY WORDS

adeno-associated virus - gene therapy - hemophilia - genetic diseases

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