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

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Abstract

Over the past decade, the Sleeping Beauty (SB) transposon system has been developed as the leading nonviral 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.

Original languageEnglish (US)
Article numberddr140
Pages (from-to)R14-R20
JournalHuman molecular genetics
Volume20
DOIs
StatePublished - Apr 2011

Bibliographical note

Funding Information:
We acknowledge the financial support of NIH grant 1R01DK082516.

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