Prolonged expression of a lysosomal enzyme in mouse liver after Sleeping Beauty transposon-mediated gene delivery: implications for non-viral gene therapy of mucopolysaccharidoses.

@article{Aronovich2007ProlongedEO,
  title={Prolonged expression of a lysosomal enzyme in mouse liver after Sleeping Beauty transposon-mediated gene delivery: implications for non-viral gene therapy of mucopolysaccharidoses.},
  author={Elena L. Aronovich and J. Bowyer Bell and Lalitha R. Belur and Robert A. Gunther and Brenda L. Koniar and David C.C. Erickson and Patricia A. Schachern and Ilze Matise and Robert S. McIvor and Chester B. Whitley and Perry B. Hackett},
  journal={The journal of gene medicine},
  year={2007},
  volume={9 5},
  pages={
          403-15
        }
}
BACKGROUND The Sleeping Beauty (SB) transposon system is a non-viral vector system that can integrate precise sequences into chromosomes. We evaluated the SB transposon system as a tool for gene therapy of mucopolysaccharidosis (MPS) types I and VII. METHODS We constructed SB transposon plasmids for high-level expression of human beta-glucuronidase (hGUSB) or alpha-L-iduronidase (hIDUA). Plasmids were delivered with and without SB transposase to mouse liver by rapid, high-volume tail-vein… CONTINUE READING
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