Springer Protocols: Abstract: 16. Liver-Directed Gene Therapy Using the Sleeping Beauty Transposon System

16. Liver-Directed Gene Therapy Using the Sleeping Beauty Transposon System

By: Lalitha R. Belur³, R. Scott McIvor³, Andrew Wilber³

Abstract

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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.

Affiliation(s): (3) Arnold and Mabel Beckman Center for Transposon Research, Gene Therapy Program, Institute of Human Genetics, and Department of Genetics, Cell Biology and Development, University of Minnesota, Minneapolis, MN

Book Title: Gene Therapy Protocols: Design and Characterization of Gene Transfer Vectors

Series: Methods in Molecular Biology | Volume: 434 | Pub. Date: Mar-07-2008 | Page Range: 267-276 | DOI: 10.1007/978-1-60327-248-3_16

Subject: Genetics/Genomics

Key Words: Gene therapy - non-viral - Sleeping Beauty - transposon - integration - liver - hydrodynamic delivery

References

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