A Modified Hyperactive Transposon Vector for Gene Therapy [Sleeping Beauty]

Applications

• Enhance transposon-mediated gene transfer for the production of transgenic cells and animals, peptide and protein production, and for use in gene therapy

Advantages

• Modified transposase alone should enhance gene transfer by at least 7-fold compared to the unmodified transposase
• Could enhance gene transfer by more than 14-fold if modified transposase is combined with transposon modifications
• Applicable in mammalian-based systems since hyperactive transposases were assessed in cultured human cells and in vivo in mice

Publications

• Zayed H, Xia L, Yerich A, Yant SR, Kay MA, Puttaraju M, McGarrity GJ, Wiest DL, McIvor RS, Tolar J, Blazar BR. Correction of DNA protein kinase deficiency by spliceosome-mediated RNA trans-splicing and sleeping beauty transposon delivery. Mol Ther. 2007 Jul;15(7):1273-9.

Scientific Background

Stable insertion of foreign DNA into the cellular genome (transformation) plays a major role in a variety of biotechnology applications. It can be used to produce transgenic cells and animals, produce peptides and proteins, and for gene therapy. Transformation can be achieved using transposable elements, which have advantages over other methods such as homologous recombination and viral-based transformation. The Sleeping Beauty transposon is the most active of the most widespread group of transposons in nature and can be applied to gene discovery in vertebrates and importantly, promotes stable in vivo delivery of therapeutic genes in adult mice.

Patents

• Published Application: 20050003542
• Issued: 7,985,739 (USA)