ArticleJournal of molecular medicine (Berlin, Germany)2011
Development of S/MAR minicircles for enhanced and persistent transgene expression in the mouse liver.
Article in Journal of molecular medicine (Berlin, Germany), 2011. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 25 papers.
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Who cites it
25 citing papers in PubMed, 67 citations in OpenAlex.
- Plasmid2MC: efficient cell-free generation of high-purity minicircle DNA for genome editing in mammalian cells.Communications biology · 2025Article
- A novel method to increase transgene expression and the stability of gene therapy-associated episomal vectors.Acta biochimica et biophysica Sinica · 2025Article
- Article
- CMV/AAT promoter of MAR-based episomal vector enhanced transgene expression in human hepatic cells.3 Biotech · 2023Article
- Episomes and Transposases-Utilities to Maintain Transgene Expression from Nonviral Vectors.Genes · 2022Review
- Minicircle Delivery to the Neural Retina as a Gene Therapy Approach.International journal of molecular sciences · 2022Review
- An episomal DNA vector platform for the persistent genetic modification of pluripotent stem cells and their differentiated progeny.Stem cell reports · 2022Article
- Comparison of promoter, DNA vector, and cationic carrier for efficient transfection of hMSCs from multiple donors and tissue sources.Molecular therapy. Nucleic acids · 2021Article
- The Landscape of Non-Viral Gene Augmentation Strategies for Inherited Retinal Diseases.International journal of molecular sciences · 2021Review
- Novel Non-integrating DNA Nano-S/MAR Vectors Restore Gene Function in Isogenic Patient-Derived Pancreatic Tumor Models.Molecular therapy. Methods & clinical development · 2020Article
- Shortened nuclear matrix attachment regions are sufficient for replication and maintenance of episomes in mammalian cells.Molecular biology of the cell · 2019Article
- Histone deacetylase inhibitors reactivate silenced transgene in vivo.Gene therapy · 2019Article
- Low-Dose Gene Therapy for Murine PKU Using Episomal Naked DNA Vectors Expressing PAH from Its Endogenous Liver Promoter.Molecular therapy. Nucleic acids · 2017Article
- Article
- Minicircle DNA Provides Enhanced and Prolonged Transgene Expression Following Airway Gene Transfer.Scientific reports · 2016Article
- Nanoparticle-based technologies for retinal gene therapy.European journal of pharmaceutics and biopharmaceutics : official journal of Arbeitsgemeinschaft fur Pharmazeutische Verfahrenstechnik e.V · 2015Review
- Sustained gene expression in the retina by improved episomal vectors.Tissue engineering. Part A · 2014Article
- Treatment of phenylketonuria using minicircle-based naked-DNA gene transfer to murine liver.Hepatology (Baltimore, Md.) · 2014Article
- S/MAR sequence confers long-term mitotic stability on non-integrating lentiviral vector episomes without selection.Nucleic acids research · 2014Article
- A review of therapeutic prospects of non-viral gene therapy in the retinal pigment epithelium.Biomaterials · 2013Review
Corrections and comments
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Authors and funding
9 authors at 3 institutions in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
We have previously described the development of a scaffold/matrix attachment region (S/MAR) episomal vector system for in vivo application and demonstrated its utility to sustain transgene expression in the mouse liver for at least 6 months following a single administration. Subsequently, we observed that transgene expression is sustained for the lifetime of the animal. The level of expression, however, does drop appreciably over time. We hypothesised that by eliminating the bacterial components in our vectors, we could improve their performance since bacterial sequences have been shown to be responsible for the immunotoxicity of the vector and the silencing of its expression when applied in vivo. We describe here the development of a minimally sized S/MAR vector, which is devoid of extraneous bacterial sequences. This minicircle vector comprises an expression cassette and an S/MAR moiety, providing higher and more sustained transgene expression for several months in the absence of selection, both in vitro and in vivo. In contrast to the expression of our original S/MAR plasmid vector, the novel S/MAR minicircle vectors mediate increased transgene expression, which becomes sustained at about twice the levels observed immediately after administration. These promising results demonstrate the utility of minimally sized S/MAR vectors for persistent, atoxic gene expression.
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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.