Sleeping Beauty transposon system

Sleeping Beauty (SB) transposon system is a non-viral vector for gene therapy.^[1]

The SB system enables high-level stable gene transfer and sustained transgene expression in multiple primary human somatic cell types. It is a highly attractive gene transfer strategy for clinical use.^[2] Sleeping Beauty transposon-based transfection is a two-component system consisting of a transposase and a transposon containing inverted repeat/direct repeat (IR/DR) sequences that result in precise integration into a TA dinucleotide. The transposon is designed with an expression cassette of interest flanked by IR/DRs, and SB transposase mediates stable integration and reliable long-term expression of the gene of interest. It has recently been demonstrated that SB efficiently mediates gene transfer and stable gene expression in human embryonic stem cells.^[3]

References

1. Aronovich, Elena L.; McIvor, R. Scott; Hackett, Perry B. (2011-04-15). "The Sleeping Beauty transposon system: a non-viral vector for gene therapy". Human Molecular Genetics. 20 (R1): R14–R20. doi:10.1093/hmg/ddr140. ISSN 0964-6906. PMC 3095056. PMID 21459777.
2. Kebriaei, Partow; Izsvák, Zsuzsanna; Narayanavari, Suneel A.; Singh, Harjeet; Ivics, Zoltán (November 2017). "Gene Therapy with the Sleeping Beauty Transposon System". Trends in genetics: TIG. 33 (11): 852–870. doi:10.1016/j.tig.2017.08.008. ISSN 0168-9525. PMID 28964527.
3. "Sign In". cshprotocols.cshlp.org. Retrieved 2024-01-27.