Journal
BIOESSAYS
Volume 32, Issue 9, Pages 756-767Publisher
WILEY
DOI: 10.1002/bies.201000027
Keywords
integration; non-viral; SB100X transposon; therapy; trial
Categories
Funding
- EU
- Deutsche Forschungsgemeinschaft [SPP1230]
- NIH [RO1CA124782, R01CA120956, 1R01DK082516]
- NATIONAL CANCER INSTITUTE [R01CA141303, R01CA124782, R01CA120956] Funding Source: NIH RePORTER
- NATIONAL INSTITUTE OF DIABETES AND DIGESTIVE AND KIDNEY DISEASES [R01DK082516] Funding Source: NIH RePORTER
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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.
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