Current Gene Therapy

Ignacio Anegon
Director INSERM UMR 1064-Center for Research in Transplantation and Immunology
CHU de Nantes. 30, boulevard


The φC31 Integrase System for Gene Therapy

Author(s): Michele P. Calos.


The φC31 integrase system represents a novel technology that opens up new possibilities for gene therapy. The φC31 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 φC31 integrase and its derivatives offer great promise for success in the near term.

Keywords: att site, genetic disease, genomic integration, liver, non-viral, phage, serine recombinase, site-specific

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Article Details

Year: 2006
Page: [633 - 645]
Pages: 13
DOI: 10.2174/156652306779010642
Price: $58