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.
Original language | English (US) |
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Pages (from-to) | 756-767 |
Number of pages | 12 |
Journal | BioEssays |
Volume | 32 |
Issue number | 9 |
DOIs | |
State | Published - Sep 2010 |
Keywords
- Integration
- Non-viral
- SB100x transposon
- Therapy
- Trial