Integration profile and safety of an adenovirus hybrid-vector utilizing hyperactive sleeping beauty transposase for somatic integration

PLoS One. 2013 Oct 4;8(10):e75344. doi: 10.1371/journal.pone.0075344. eCollection 2013.

Abstract

We recently developed adenovirus/transposase hybrid-vectors utilizing the previously described hyperactive Sleeping Beauty (SB) transposase HSB5 for somatic integration and we could show stabilized transgene expression in mice and a canine model for hemophilia B. However, the safety profile of these hybrid-vectors with respect to vector dose and genotoxicity remains to be investigated. Herein, we evaluated this hybrid-vector system in C57Bl/6 mice with escalating vector dose settings. We found that in all mice which received the hyperactive SB transposase, transgene expression levels were stabilized in a dose-dependent manner and that the highest vector dose was accompanied by fatalities in mice. To analyze potential genotoxic side-effects due to somatic integration into host chromosomes, we performed a genome-wide integration site analysis using linker-mediated PCR (LM-PCR) and linear amplification-mediated PCR (LAM-PCR). Analysis of genomic DNA samples obtained from HSB5 treated female and male mice revealed a total of 1327 unique transposition events. Overall the chromosomal distribution pattern was close-to-random and we observed a random integration profile with respect to integration into gene and non-gene areas. Notably, when using the LM-PCR protocol, 27 extra-chromosomal integration events were identified, most likely caused by transposon excision and subsequent transposition into the delivered adenoviral vector genome. In total, this study provides a careful evaluation of the safety profile of adenovirus/Sleeping Beauty transposase hybrid-vectors. The obtained information will be useful when designing future preclinical studies utilizing hybrid-vectors in small and large animal models.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adenoviridae / genetics*
  • Animals
  • Computational Biology
  • Enzyme-Linked Immunosorbent Assay
  • Genetic Vectors / genetics*
  • HeLa Cells
  • Humans
  • Mice
  • Mice, Inbred C57BL
  • Transposases / genetics*

Substances

  • Transposases

Grants and funding

This work was supported in part by DFG grant SPP 1230, the Wilhelm Sander-Foundation, EU Framework Programme 7 (Persistent Transgenesis), and the Friedrich-Baur-Foundation (A.E.). W.Z. was supported by a fellowship from the Chinese Scholarship Council (CSC) in cooperation with Northwest A&F University, Yangling, China. The funders had no role in study design, data collection and analysis, decision to publish, or preparation of the manuscript.