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      The phiC31 integrase system for gene therapy.

      1
      Current gene therapy
      Bentham Science Publishers Ltd.

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          Abstract

          The phiC31 integrase system represents a novel technology that opens up new possibilities for gene therapy. The phiC31 integrase can integrate introduced plasmid DNA into preferred locations in unmodified mammalian genomes, resulting in robust, long-term expression of the integrated transgene. This review describes the nature of the integration reaction and the genomic integration sites used by the enzyme in human cells. Preclinical applications of the system to gene therapy to date are summarized, including in vivo use in liver, muscle, eye, and joint and ex vivo use in skin keratinocytes, muscle precursor cells, and T cell lines. The safety of this phage integrase system for gene therapy is evaluated, and its strengths and limitations are compared to other gene therapy approaches. Ongoing and planned improvements to the phage integrase system are discussed. We conclude that gene therapy strategies using phiC31 integrase and its derivatives offer great promise for success in the near term.

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          Author and article information

          Journal
          Curr Gene Ther
          Current gene therapy
          Bentham Science Publishers Ltd.
          1566-5232
          1566-5232
          Dec 2006
          : 6
          : 6
          Affiliations
          [1 ] Department of Genetics, Stanford University School of Medicine, Stanford, CA 94305-5120, USA. calos@stanford.edu
          Article
          10.2174/156652306779010642
          17168696
          dc785b86-44ed-40fb-9081-50fbb14283c9
          History

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