Somatic integration and long-term transgene expression in normal and haemophilic mice using a DNA transposon system View Full Text


Ontology type: schema:ScholarlyArticle     


Article Info

DATE

2000-05

AUTHORS

Stephen R. Yant, Leonard Meuse, Winnie Chiu, Zoltan Ivics, Zsuzsanna Izsvak, Mark A. Kay

ABSTRACT

The development of non-viral gene-transfer technologies that can support stable chromosomal integration and persistent gene expression in vivo is desirable. Here we describe the successful use of transposon technology for the nonhomologous insertion of foreign genes into the genomes of adult mammals using naked DNA. We show that the Sleeping Beauty transposase can efficiently insert transposon DNA into the mouse genome in approximately 5-6% of transfected mouse liver cells. Chromosomal transposition resulted in long-term expression (>5 months) of human blood coagulation factor IX at levels that were therapeutic in a mouse model of haemophilia B. Our results establish DNA-mediated transposition as a new genetic tool for mammals, and provide new strategies to improve existing non-viral and viral vectors for human gene therapy applications. More... »

PAGES

ng0500_35

Journal

TITLE

Nature Genetics

ISSUE

1

VOLUME

25

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  • Identifiers

    URI

    http://scigraph.springernature.com/pub.10.1038/75568

    DOI

    http://dx.doi.org/10.1038/75568

    DIMENSIONS

    https://app.dimensions.ai/details/publication/pub.1039686973

    PUBMED

    https://www.ncbi.nlm.nih.gov/pubmed/10802653


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