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Translating Sleeping Beauty transposition into cellular therapies: Victories and challenges

Item Type:Review
Title:Translating Sleeping Beauty transposition into cellular therapies: Victories and challenges
Creators Name:Izsvak, Z., Hackett, P.B., Cooper, L.J. and Ivics, Z.
Abstract:Recent results confirm that long-term expression of therapeutic transgenes can be achieved by using a transposon-based system in primary stem cells and in vivo. Transposable elements are natural DNA transfer vehicles that are capable of efficient genomic insertion. The latest generation, Sleeping Beauty transposon-based hyperactive vector (SB100X), is able to address the basic problem of non-viral approaches - that is, low efficiency of stable gene transfer. The combination of transposon-based non-viral gene transfer with the latest improvements of non-viral delivery techniques could provide a long-term therapeutic effect without compromising biosafety. The new challenges of pre-clinical research will focus on further refinement of the technology in large animal models and improving the safety profile of SB vectors by target-selected transgene integration into genomic "safe harbors." The first clinical application of the SB system will help to validate the safety of this approach.
Keywords:Integration, Non-Viral, SB100X Transposon, Therapy, Trial, Animals
Page Range:756-767
Date:September 2010
Additional Information:Erratum in: BioEssays 33(6): 478-479.
Official Publication:https://doi.org/10.1002/bies.201000027
PubMed:View item in PubMed

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