Translating Sleeping Beauty transposition into cellular therapies: Victories and challenges

Zsuzsanna Izsvák, Perry B. Hackett, Laurence J.N. Cooper, Zoltán Ivics

Research output: Contribution to journalReview articlepeer-review

79 Scopus citations

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 languageEnglish (US)
Pages (from-to)756-767
Number of pages12
JournalBioEssays
Volume32
Issue number9
DOIs
StatePublished - Sep 2010

Keywords

  • Integration
  • Non-viral
  • SB100x transposon
  • Therapy
  • Trial

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