ArticleMolecular therapy : the journal of the American Society of Gene Therapy2012
Retargeting sleeping beauty transposon insertions by engineered zinc finger DNA-binding domains.
Article in Molecular therapy : the journal of the American Society of Gene Therapy, 2012. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 43 papers.
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Who cites it
43 citing papers in PubMed, 65 citations in OpenAlex.
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- Exploration of the potential of genomic editing in the treatment of congenital adrenal hyperplasia.Frontiers in endocrinology · 2025Review
- Targeted gene delivery systems for T-cell engineering.Cellular oncology (Dordrecht, Netherlands) · 2024Review
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- Current strategies employed in the manipulation of gene expression for clinical purposes.Journal of translational medicine · 2022Review
- Episomes and Transposases-Utilities to Maintain Transgene Expression from Nonviral Vectors.Genes · 2022Review
- Engineered Sleeping Beauty transposase redirects transposon integration away from genes.Nucleic acids research · 2022Article
- Jumping Ahead withViruses · 2021Review
- RNA-guided retargeting of SeLife · 2020Article
- Gene Editing and Genotoxicity: Targeting the Off-Targets.Frontiers in genome editing · 2020Review
- Jump around: transposons in and out of the laboratory.F1000Research · 2020Review
- Preclinical Evaluation of a Cell-Based Gene Therapy Using the Sleeping Beauty Transposon System in Choroidal Neovascularization.Molecular therapy. Methods & clinical development · 2019Article
- A highly soluble Sleeping Beauty transposase improves control of gene insertion.Nature biotechnology · 2019Article
- Self-assembled peptide-poloxamine nanoparticles enable in vitro and in vivo genome restoration for cystic fibrosis.Nature nanotechnology · 2019Article
- Chimeric Antigen Receptor Library Screening Using a Novel NF-κB/NFAT Reporter Cell Platform.Molecular therapy : the journal of the American Society of Gene Therapy · 2019Article
- Targeted gene knockin in zebrafish using the 28S rDNA-specific non-LTR-retrotransposon R2Ol.Mobile DNA · 2019Article
- Efficient Non-viral Gene Delivery into Human Hematopoietic Stem Cells by Minicircle Sleeping Beauty Transposon Vectors.Molecular therapy : the journal of the American Society of Gene Therapy · 2018Article
- Article
Corrections and comments
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Authors and funding
7 authors at 3 institutions in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
The Sleeping Beauty (SB) transposon is a nonviral, integrating vector system with proven efficacy in preclinical animal models, and thus holds promise for future clinical applications. However, SB has a close-to-random insertion profile that could lead to genotoxic effects, thereby presenting a potential safety issue. We evaluated zinc finger (ZF) DNA-binding domains (DBDs) for their abilities to introduce a bias into SB's insertion profile. E2C, that binds a unique site in the erbB-2 gene, mediated locus-specific transposon insertions at low frequencies. A novel ZF targeting LINE1 repeats, ZF-B, showed specific binding to an 18-bp site represented by ~12,000 copies in the human genome. We mapped SB insertions using linear-amplification (LAM)-PCR and Illumina sequencing. Targeted insertions with ZF-B peaked at approximately fourfold enrichment of transposition around ZF-B binding sites yielding ~45% overall frequency of insertion into LINE1. A decrease in the ZF-B dataset with respect to transposon insertions in genes was found, suggesting that LINE1 repeats act as a sponge that "soak up" a fraction of SB insertions and thereby redirect them away from genes. Improvements in ZF technology and a careful choice of targeted genomic regions may improve the safety profile of SB for future clinical applications.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.