ArticleMolecular therapy. Nucleic acids2013
Efficient sleeping beauty DNA transposition from DNA minicircles.
Article in Molecular therapy. Nucleic acids, 2013. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 16 papers.
What it found
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The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
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Who cites it
16 citing papers in PubMed.
- CARAMBA: a first-in-human clinical trial with SLAMF7 CAR-T cells prepared by virus-free Sleeping Beauty gene transfer to treat multiple myeloma.Gene therapy · 2021Trial
- Engineering of BZ transposase and transposon donor vector for enhanced efficiency and safety in gene delivery applications.Nucleic acids research · 2025Article
- Emerging CAR immunotherapies: broadening therapeutic horizons beyond cancer.Clinical and experimental medicine · 2025Review
- Improving cell and gene therapy safety and performance using next-generation Nanoplasmid vectors.Molecular therapy. Nucleic acids · 2023Review
- Life-threatening viral disease in a novel form of autosomal recessive IFNAR2 deficiency in the Arctic.The Journal of experimental medicine · 2022Article
- The Past, Present, and Future of Non-Viral CAR T Cells.Frontiers in immunology · 2022Review
- Enhanced Tailored MicroRNA Sponge Activity of RNA Pol II-Transcribed TuD Hairpins Relative to Ectopically Expressed ciRS7-Derived circRNAs.Molecular therapy. Nucleic acids · 2018Article
- 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
- Preclinical and clinical advances in transposon-based gene therapy.Bioscience reports · 2017Review
- Enhanced CAR T-cell engineering using non-viral Sleeping Beauty transposition from minicircle vectors.Leukemia · 2017Article
- Retroviral vectors and transposons for stable gene therapy: advances, current challenges and perspectives.Journal of translational medicine · 2016Review
- piggyBac transposons expressing full-length human dystrophin enable genetic correction of dystrophic mesoangioblasts.Nucleic acids research · 2016Article
- Neuroprotective therapies in glaucoma: II. Genetic nanotechnology tools.Frontiers in neuroscience · 2015Review
- DNA transposition by protein transduction of the piggyBac transposase from lentiviral Gag precursors.Nucleic acids research · 2014Article
- Driving DNA transposition by lentiviral protein transduction.Mobile genetic elements · 2014Article
- DNA transposon-based gene vehicles - scenes from an evolutionary drive.Journal of biomedical science · 2013Review
Corrections and comments
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Authors and funding
6 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
DNA transposon-based vectors have emerged as new potential delivery tools in therapeutic gene transfer. Such vectors are now showing promise in hematopoietic stem cells and primary human T cells, and clinical trials with transposon-engineered cells are on the way. However, the use of plasmid DNA as a carrier of the vector raises safety concerns due to the undesirable administration of bacterial sequences. To optimize vectors based on the Sleeping Beauty (SB) DNA transposon for clinical use, we examine here SB transposition from DNA minicircles (MCs) devoid of the bacterial plasmid backbone. Potent DNA transposition, directed by the hyperactive SB100X transposase, is demonstrated from MC donors, and the stable transfection rate is significantly enhanced by expressing the SB100X transposase from MCs. The stable transfection rate is inversely related to the size of circular donor, suggesting that a MC-based SB transposition system benefits primarily from an increased cellular uptake and/or enhanced expression which can be observed with DNA MCs. DNA transposon and transposase MCs are easily produced, are favorable in size, do not carry irrelevant DNA, and are robust substrates for DNA transposition. In accordance, DNA MCs should become a standard source of DNA transposons not only in therapeutic settings but also in the daily use of the SB system.Molecular Therapy - Nucleic Acids (2013) 2, e74; doi:10.1038/mtna.2013.1; published online 26 February 2013.
Identifiers
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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.