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  • Title: Site-specific genomic integration produces therapeutic Factor IX levels in mice.
    Author: Olivares EC, Hollis RP, Chalberg TW, Meuse L, Kay MA, Calos MP.
    Journal: Nat Biotechnol; 2002 Nov; 20(11):1124-8. PubMed ID: 12379870.
    Abstract:
    We used the integrase from phage phiC31 to integrate the human Factor IX (hFIX) gene permanently into specific sites in the mouse genome. A plasmid containing attB and an expression cassette for hFIX was delivered to the livers of mice by using high-pressure tail vein injection. When an integrase expression plasmid was co-injected, hFIX serum levels increased more than tenfold to approximately 4 microg/ml, similar to normal FIX levels, and remained stable throughout the more than eight months of the experiment. hFIX levels persisted after partial hepatectomy, suggesting genomic integration of the vector. Site-specific integration was proven by characterizing and quantifying genomic integration in the liver at the DNA level. Integration was documented at two pseudo-attP sites, native sequences with partial identity to attP, with one site highly predominant. This study demonstrates in vivo gene transfer in an animal by site-specific genomic integration.
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