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| 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 |
| Source: | BioEssays |
| ISSN: | 0265-9247 |
| Publisher: | Wiley-Blackwell |
| Volume: | 32 |
| Number: | 9 |
| 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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