ArticleGene therapy2011
Non-viral S/MAR vectors replicate episomally in vivo when provided with a selective advantage.
Article in Gene therapy, 2011. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 23 papers.
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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
23 citing papers in PubMed, 44 citations in OpenAlex.
- Regulatory T Cell-Based Adoptive Cell Therapy in Autoimmunity.International journal of molecular sciences · 2025Review
- In Vivo Selection of S/MAR Sequences to Favour AAV Episomal Maintenance in Dividing Cells.International journal of molecular sciences · 2024Article
- Development of Cationic Lipid LAH4-L1 siRNA Complexes for Focused Ultrasound Enhanced Tumor Uptake.Molecular pharmaceutics · 2023Article
- Development of an Orthotopic HPV16-Dependent Base of Tongue Tumor Model in MHC-Humanized Mice.Pathogens (Basel, Switzerland) · 2023Article
- An episomal DNA vector platform for the persistent genetic modification of pluripotent stem cells and their differentiated progeny.Stem cell reports · 2022Article
- Hypoxia-sensitive miRNA regulationFrontiers in cell and developmental biology · 2022Article
- Understanding In Vivo Fate of Nucleic Acid and Gene Medicines for the Rational Design of Drugs.Pharmaceutics · 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
- The IS2 Element Improves Transcription Efficiency of Integration-Deficient Lentiviral Vector Episomes.Molecular therapy. Nucleic acids · 2018Article
- Identification of a potent MAR element from the human genome and assessment of its activity in stably transfected CHO cells.Journal of cellular and molecular medicine · 2018Article
- Progresses towards safe and efficient gene therapy vectors.Oncotarget · 2015Review
- Sustained gene expression in the retina by improved episomal vectors.Tissue engineering. Part A · 2014Article
- Long-term episomal transgene expression from mitotically stable integration-deficient lentiviral vectors.Human gene therapy · 2014Article
- S/MAR sequence confers long-term mitotic stability on non-integrating lentiviral vector episomes without selection.Nucleic acids research · 2014Article
- Persistence of non-viral vector mediated RPE65 expression: case for viability as a gene transfer therapy for RPE-based diseases.Journal of controlled release : official journal of the Controlled Release Society · 2013Article
- A review of therapeutic prospects of non-viral gene therapy in the retinal pigment epithelium.Biomaterials · 2013Review
- Genetic modification of dividing cells using episomally maintained S/MAR DNA vectors.Molecular therapy. Nucleic acids · 2013Article
- Generation of a tumor- and tissue-specific episomal non-viral vector system.BMC biotechnology · 2013Article
- S/MAR-containing DNA nanoparticles promote persistent RPE gene expression and improvement in RPE65-associated LCA.Human molecular genetics · 2013Article
- Article
Corrections and comments
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Authors and funding
4 authors at 1 institution in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
The ideal gene therapy vector should enable persistent expression without the limitations of safety and reproducibility. We previously reported that a prototype plasmid vector, containing a scaffold matrix attachment region (S/MAR) domain and the luciferase reporter gene, showed transgene expression for at least 6 months following a single administration to MF1 mice. Following partial hepatectomy of the animals, however, we found no detectable vector replication and subsequent propagation in vivo. To overcome this drawback, we have now developed an in vivo liver selection strategy by which liver cells transfected with an S/MAR plasmid are provided with a survival advantage over non-transfected cells. This allows an enrichment of vectors that are capable of replicating and establishing themselves as extra-chromosomal entities in the liver. Accordingly, a novel S/MAR plasmid encoding the Bcl-2 gene was constructed; Bcl-2 expression confers resistance against apoptosis-mediated challenges by the Fas-activating antibody Jo2. Following hydrodynamic delivery to the livers of mice and frequent Jo2 administrations, we demonstrate that this Bcl-luciferase S/MAR plasmid is indeed capable of providing sustained luciferase reporter gene expression for over 3 months and that this plasmid replicates as an episomal entity in vivo. These results provide proof-of-principle that S/MAR vectors are capable of preventing transgene silencing, are resistant to integration and are able to confer mitotic stability in vivo when provided with a selective advantage.
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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.