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| Item Type: | Review |
|---|---|
| Title: | Sleeping Beauty transposon vectors for therapeutic applications: advances and challenges |
| Creators Name: | Narayanavari, S.A. and Izsvák, Z. |
| Abstract: | Transposable elements are natural, non-viral gene delivery vehicles capable of mediating stable genomic integration. The Sleeping Beauty (SB) transposon has the ability to cut-and-paste the 'gene of interest' into the genome, providing the basis for long-term, permanent transgene expression in transgenic cells and organisms. The SB transposon system is relatively well characterized, and has been extensively engineered for efficient gene delivery and gene discovery purposes in a wide range of vertebrates, including humans. The SB system is a safe and simple-to-use vector that enables cost-effective, rapid preparation of therapeutic doses of cell products. Recently, there has been a growing interest in using the SB system for therapy as evidenced by the large number of pre-clinical studies. SB moved swiftly from pre-clinical to clinical trials in almost a decade. In this article, we highlight the advancements and challenges associated with the SB system in various therapeutic applications. We also provide an overview that has been exploited by spin-off companies based on the SB system. |
| Source: | Cell and Gene Therapy Insights |
| ISSN: | 2059-7800 |
| Publisher: | Bioinsights |
| Volume: | 3 |
| Number: | 2 |
| Page Range: | 131-158 |
| Date: | 30 March 2017 |
| Official Publication: | https://doi.org/10.18609/cgti.2017.014 |
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