Module 8 : GENE THERAPY : INTRODUCTION AND METHODS

Lecture 1 : Gene Therapy: Introduction and Methods

•  Sleeping Beauty Transposition

This is a non viral method of gene transfer which offer the advantage of stable DNA integration into the chromosomes of vertebrates. The sleeping beauty transposition system consists of a sleeping beauty transposon and a sleeping beauty transposase. The SB transposon consists of two terminal inverted repeats present each at the ends of the gene of interest and the SB transposase mediates excision of the SB transposon and its integration into a site of chromosome having a dinucleotide dimer TA by cut and paste mechanism.

Figure 8-1.7.2.6: Cut and paste mechanism of a DNA transposon by a transposase (txp gene)

Adapted and modified from:

http://www.discoverygenomics.net/pdf/SBT_ProlongedExpressions.pdf

For gene therapy transposase gene is replaced by our gene of interest and transposase is provided in trans as shown in Figure 8-1.7.2.7.