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 Scopus citations

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

Keywords

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

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