Liver-directed gene therapy using the sleeping beauty transposon system

Lalitha Belur, R S Mc Ivor, Andrew Wilber

Research output: Chapter in Book/Report/Conference proceedingChapter

14 Citations (Scopus)

Abstract

Sleeping Beauty (SB) is a transposon system genetically reconstructed from teleost fish that mediates chromosomal integration of DNA sequences by a cut-and-paste mechanism. SB has been shown to mediate transposition in a variety of cells and tissues, has been used for the generation of transgenic animals and has been tested as a vector for gene therapy in several animal models of human disease. Here, we describe methods that we have developed for testing SB-mediated transposition, first in cultured mammalian cells, and then in vivo using a combination of rapid, high-volume tail vein injection for DNA delivery to the liver along with in vivo bioluminescence imaging to monitor sustained luciferase gene expression in individual animals.

Original languageEnglish (US)
Title of host publicationGene Therapy Protocols
Subtitle of host publicationDesign and Characterization of Gene Transfer Vectors
PublisherHumana Press
Pages267-276
Number of pages10
Volume434
ISBN (Print)9781603272476
DOIs
StatePublished - Sep 16 2008

Publication series

NameMethods in Molecular Biology
Volume434
ISSN (Print)1064-3745

Fingerprint

Beauty
Genetic Therapy
Liver
Animal Disease Models
Genetically Modified Animals
Ointments
Luciferases
Tail
Veins
Cultured Cells
Fishes
Gene Expression
Injections
DNA

Keywords

  • Gene therapy
  • Hydrodynamic delivery
  • Integration
  • Liver
  • Non-viral
  • Sleeping Beauty
  • Transposon

Cite this

Belur, L., Mc Ivor, R. S., & Wilber, A. (2008). Liver-directed gene therapy using the sleeping beauty transposon system. In Gene Therapy Protocols: Design and Characterization of Gene Transfer Vectors (Vol. 434, pp. 267-276). (Methods in Molecular Biology; Vol. 434). Humana Press. https://doi.org/10.1007/978-1-60327-248-3_16

Liver-directed gene therapy using the sleeping beauty transposon system. / Belur, Lalitha; Mc Ivor, R S; Wilber, Andrew.

Gene Therapy Protocols: Design and Characterization of Gene Transfer Vectors. Vol. 434 Humana Press, 2008. p. 267-276 (Methods in Molecular Biology; Vol. 434).

Research output: Chapter in Book/Report/Conference proceedingChapter

Belur, L, Mc Ivor, RS & Wilber, A 2008, Liver-directed gene therapy using the sleeping beauty transposon system. in Gene Therapy Protocols: Design and Characterization of Gene Transfer Vectors. vol. 434, Methods in Molecular Biology, vol. 434, Humana Press, pp. 267-276. https://doi.org/10.1007/978-1-60327-248-3_16
Belur L, Mc Ivor RS, Wilber A. Liver-directed gene therapy using the sleeping beauty transposon system. In Gene Therapy Protocols: Design and Characterization of Gene Transfer Vectors. Vol. 434. Humana Press. 2008. p. 267-276. (Methods in Molecular Biology). https://doi.org/10.1007/978-1-60327-248-3_16
Belur, Lalitha ; Mc Ivor, R S ; Wilber, Andrew. / Liver-directed gene therapy using the sleeping beauty transposon system. Gene Therapy Protocols: Design and Characterization of Gene Transfer Vectors. Vol. 434 Humana Press, 2008. pp. 267-276 (Methods in Molecular Biology).
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