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piggyBac as a high-capacity transgenesis and gene-therapy vector in human cells and mice.

Publication ,  Journal Article
Li, R; Zhuang, Y; Han, M; Xu, T; Wu, X
Published in: Dis Model Mech
May 2013

The stable genomic integration and expression of a large transgene is a major hurdle in gene therapy. We show that the modified piggyBac (PB) transposon system can be used to introduce a 207 kb genomic DNA fragment containing the RORγ/γt locus into human cells and mice. PB-mediated transgenesis results in a single copy of a stably inherited and expressed transgene. These results indicate that PB could serve as an effective high-capacity vector for functional analysis of the mammalian genome and for gene therapy in human cells.

Duke Scholars

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Published In

Dis Model Mech

DOI

EISSN

1754-8411

Publication Date

May 2013

Volume

6

Issue

3

Start / End Page

828 / 833

Location

England

Related Subject Headings

  • Transgenes
  • Mutagenesis, Insertional
  • Molecular Sequence Data
  • Mice
  • Humans
  • HEK293 Cells
  • Genetic Vectors
  • Genetic Therapy
  • Gene Transfer Techniques
  • Embryonic Stem Cells
 

Citation

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ICMJE
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Li, R., Zhuang, Y., Han, M., Xu, T., & Wu, X. (2013). piggyBac as a high-capacity transgenesis and gene-therapy vector in human cells and mice. Dis Model Mech, 6(3), 828–833. https://doi.org/10.1242/dmm.010827
Li, Rongbo, Yuan Zhuang, Min Han, Tian Xu, and Xiaohui Wu. “piggyBac as a high-capacity transgenesis and gene-therapy vector in human cells and mice.Dis Model Mech 6, no. 3 (May 2013): 828–33. https://doi.org/10.1242/dmm.010827.
Li R, Zhuang Y, Han M, Xu T, Wu X. piggyBac as a high-capacity transgenesis and gene-therapy vector in human cells and mice. Dis Model Mech. 2013 May;6(3):828–33.
Li, Rongbo, et al. “piggyBac as a high-capacity transgenesis and gene-therapy vector in human cells and mice.Dis Model Mech, vol. 6, no. 3, May 2013, pp. 828–33. Pubmed, doi:10.1242/dmm.010827.
Li R, Zhuang Y, Han M, Xu T, Wu X. piggyBac as a high-capacity transgenesis and gene-therapy vector in human cells and mice. Dis Model Mech. 2013 May;6(3):828–833.
Journal cover image

Published In

Dis Model Mech

DOI

EISSN

1754-8411

Publication Date

May 2013

Volume

6

Issue

3

Start / End Page

828 / 833

Location

England

Related Subject Headings

  • Transgenes
  • Mutagenesis, Insertional
  • Molecular Sequence Data
  • Mice
  • Humans
  • HEK293 Cells
  • Genetic Vectors
  • Genetic Therapy
  • Gene Transfer Techniques
  • Embryonic Stem Cells