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Hum Mol Genet. 2011 Apr 15;20(R1):R14-20. doi: 10.1093/hmg/ddr140. Epub 2011 Apr 1.

The Sleeping Beauty transposon system: a non-viral vector for gene therapy.

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  • 1Department of Genetics, Cell Biology and Development, The Center for Genome Engineering, Institute of Human Genetics, University of Minnesota, 6-160 Jackson Hall, 321 Church St. SE, Minneapolis, MN 55455, USA. arono001@umn.edu

Abstract

Over the past decade, the Sleeping Beauty (SB) transposon system has been developed as the leading non-viral vector for gene therapy. This vector combines the advantages of viruses and naked DNA. Here we review progress over the last 2 years in vector design, methods of delivery and safety that have supported its use in the clinic. Currently, the SB vector has been validated for ex vivo gene delivery to stem cells, including T-cells for the treatment of lymphoma. Progress in delivery of SB transposons to liver for treatment of various systemic diseases, such as hemophilia and mucopolysaccharidoses types I and VII, has encountered some problems, but even here progress is being made.

PMID:
21459777
[PubMed - indexed for MEDLINE]
PMCID:
PMC3095056
Free PMC Article

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