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- W2085206589 abstract "The phiC31 integrase system effectively integrates plasmid DNA carrying an attB site into a limited number of endogenous pseudo attP sites in mammalian genomes, leading to robust, long_term expression of transgenes. The phiC31 system has been used to genetically engineer Drosophila and Xenopus and for in vivo and ex vivo gene therapy in several tissues in model organisms including mouse, rat, and rabbit. The integrase system appears to be well suited for long_term gene therapy in humans. In particular, the phiC31 system may allow long_term gene expression in hepatocytes in vivo. A simple and efficient delivery system for liver using naked DNA is available for mice by using high_pressure tail vein injection. With this delivery method and phiC31 integrase, we demonstrated lifelong, therapeutic levels of expression of human factor IX in mice. An adapted version of this DNA delivery method may be appropriate for use in patients. Our current study addressed the amount of liver perturbation and subsequent proliferation seen after the high_pressure tail vein procedure. Using a BrdU analog to label proliferating hepatocytes, we showed that a sizable fraction of the hepatocytes entered S phase within two days after injection. By one week post_injection, however, hepatocytes had stopped proliferating and the livers appeared normal by histological analysis. We also determined the immunogenicity and ability to re_deliver phiC31 integrase by measurement of expression levels of a reporter gene after repeated doses. The level and duration of gene expression achievable with the phiC31 integrase system in liver makes it an attractive strategy for clinical development of gene therapies for diseases such as hemophilia. Michele Calos is a co_founder and consultant for Poetic Genetics, LLC, which has licensed the phiC31 integrase technology." @default.
- W2085206589 created "2016-06-24" @default.
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- W2085206589 date "2006-01-01" @default.
- W2085206589 modified "2023-10-17" @default.
- W2085206589 title "863. Liver Gene Therapy for Hemophilia with phiC31 Integrase" @default.
- W2085206589 doi "https://doi.org/10.1016/j.ymthe.2006.08.949" @default.
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