Hyperactive Piggybac Transposons for Sustained and Robust Liver-targeted Gene Therapy Authors
نویسندگان
چکیده
Department of Gene Therapy & Regenerative Medicine, Free University of Brussels, Brussels, Belgium & Center for Molecular & Vascular Biology, Department of Cardiovascular Medicine, University of Leuven, Leuven, Belgium; Molecular Immunology Unit, Institute of Child Health, University College London, London, United Kingdom; Institute for Women’s Health Gene Transfer Technology Group, University College London, London; School of Pathology, Faculty of Health Sciences, University of the Witwatersrand, South Africa; University of Surrey, Molecular Medicine Group, Surrey, UK.
منابع مشابه
Hyperactive PiggyBac Transposons for Sustained and Robust Liver-targeted Gene Therapy
The development of robust nonviral vectors could facilitate clinical gene therapy applications and may overcome some of the immune complications of viral vectors. Nevertheless, most nonviral gene deliver approaches typically yield only transient and/or low gene expression. To address these caveats, we have explored piggyBac transposons to correct hemophilia B by liver-directed factor IX (FIX) g...
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Effective gene therapy requires robust delivery of the desired genes into the relevant target cells, long-term gene expression, and minimal risks of secondary effects. The development of efficient and safe nonviral vectors would greatly facilitate clinical gene therapy studies. However, nonviral gene transfer approaches typically result in only limited stable gene transfer efficiencies in most ...
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DNA transposons have been widely used for transgenesis and insertional mutagenesis in various organisms. Among the transposons active in mammalian cells, the moth-derived transposon piggyBac is most promising with its highly efficient transposition, large cargo capacity, and precise repair of the donor site. Here we report the generation of a hyperactive piggyBac transposase. The active transpo...
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BACKGROUND DNA transposons have emerged as indispensible tools for manipulating vertebrate genomes with applications ranging from insertional mutagenesis and transgenesis to gene therapy. To fully explore the potential of two highly active DNA transposons, piggyBac and Tol2, as mammalian genetic tools, we have conducted a side-by-side comparison of the two transposon systems in the same setting...
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We characterized a recently developed hyperactive piggyBac (pB) transposase enzyme [containing seven mutations (7pB)] for gene transfer in human cells in vitro and to somatic cells in mice in vivo. Despite a protein level expression similar to that of native pB, 7pB significantly increased the gene transfer efficiency of a neomycin resistance cassette transposon in both HEK293 and HeLa cultured...
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