Hyperactive sleeping beauty transposase enables persistent phenotypic correction in mice and a canine model for hemophilia B.

@article{Hausl2010HyperactiveSB,
  title={Hyperactive sleeping beauty transposase enables persistent phenotypic correction in mice and a canine model for hemophilia B.},
  author={Martin A Hausl and Wenli Zhang and Nadine Muether and Christina Theresa Rauschhuber and Helen W G Franck and Elizabeth P. Merricks and Timothy C Nichols and Mark A. Kay and Anja Ehrhardt},
  journal={Molecular therapy : the journal of the American Society of Gene Therapy},
  year={2010},
  volume={18 11},
  pages={1896-906}
}
Sleeping Beauty (SB) transposase enables somatic integration of exogenous DNA in mammalian cells, but potency as a gene transfer vector especially in large mammals has been lacking. Herein, we show that hyperactive transposase system delivered by high-capacity adenoviral vectors (HC-AdVs) can result in somatic integration of a canine factor IX (cFIX) expression-cassette in canine liver, facilitating stabilized transgene expression and persistent haemostatic correction of canine hemophilia B… CONTINUE READING
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