Ontology highlight
ABSTRACT:
SUBMITTER: Aronovich EL
PROVIDER: S-EPMC2835207 | biostudies-literature | 2009 Jul
REPOSITORIES: biostudies-literature
Aronovich Elena L EL Bell Jason B JB Khan Shaukat A SA Belur Lalitha R LR Gunther Roland R Koniar Brenda B Schachern Patricia A PA Parker Josh B JB Carlson Cathy S CS Whitley Chester B CB McIvor R Scott RS Gupta Pankaj P Hackett Perry B PB
Molecular therapy : the journal of the American Society of Gene Therapy 20090421 7
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 le ...[more]