Systemic correction of storage disease in MPS I NOD/SCID mice using the sleeping beauty transposon system.

@article{Aronovich2009SystemicCO,
  title={Systemic correction of storage disease in MPS I NOD/SCID mice using the sleeping beauty transposon system.},
  author={Elena L. Aronovich and J. Bowyer Bell and Shaukat Ali Khan and Lalitha R. Belur and Robert A. Gunther and Brenda L. Koniar and Patricia A. Schachern and Josh Berken Parker and Cathy S Carlson and Chester B. Whitley and Robert S. McIvor and Piyush Gupta and Perry B. Hackett},
  journal={Molecular therapy : the journal of the American Society of Gene Therapy},
  year={2009},
  volume={17 7},
  pages={
          1136-44
        }
}
The Sleeping Beauty (SB) transposon system is a nonviral vector that directs transgene integration into vertebrate genomes. We hydrodynamically delivered SB transposon plasmids encoding human alpha-L-iduronidase (hIDUA) at two DNA doses, with and without an SB transposase gene, to NOD.129(B6)-Prkdc(scid) IDUA(tm1Clk)/J mice. In transposon-treated, nonobese diabetic/severe combined immunodeficiency (NOD/SCID) mice with mucopolysaccharidosis type I (MPS I), plasma IDUA persisted for 18 weeks at… CONTINUE READING
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