The DOI matches its Crossref record; 24 of the 24 references it cites were found in a registry or an index.
Minicircle DNA vectors devoid of bacterial DNA result in persistent and high-level transgene expression in vivo
Open accessAbstract
The loss of transgene expression has been a major obstacle to the development of nonviral vectors for the treatment of human diseases. We previously demonstrated that bacterial DNA linked to a mammalian expression cassette resulted in transcriptional silencing of the transgene in vivo. To confirm these studies and develop a means to produce a robust DNA vector that is not silenced in vivo, we developed a phage phiC31 integrase-mediated intramolecular recombination technology to prepare minicircle vector DNA devoid of the bacterial backbone and then compared the transgene expression profile of the minicircle with different molecular forms of plasmid DNAs in mice. We demonstrate that minicircular DNAs devoid of bacterial sequences expressed 45- and 560-fold more serum human factor IX and alpha1-antitrypsin, respectively, compared to standard plasmid DNAs transfected into mouse liver. Our data suggest that minicircles are capable of expressing high and persistent levels of therapeutic products in vivo and have a great potential to serve as episomal vectors for the treatment of a wide variety of diseases.
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Cites
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Self-citationA phage integrase directs efficient site-specific integration in human cells
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Cited by
In vivo genome editing via CRISPR/Cas9 mediated homology-independent targeted integration
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A nonviral minicircle vector for deriving human iPS cells
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Self-citationNonviral Gene Delivery: Principle, Limitations, and Recent Progress
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State-of-the-art gene-based therapies: the road ahead
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Self-citationNew Vaccine Technologies to Combat Outbreak Situations
Rauch, S., Jasny, E., Schmidt, K. E., & Petsch, B. (2018) · Frontiers in Immunology · doi:10.3389/fimmu.2018.01963 · Cited by 579
Metadata
- Authors
- Chen, Z.-Y., He, C.-Y., Ehrhardt, A., & Kay, M. A.
- Venue
- Molecular Therapy
- Publisher
- Elsevier BV
- Published
- 2003-09
- Volume
- 8
- Issue
- 3
- Pages
- 495-500
- ISSN
- 1525-0016 (ISSN-L)
- OpenAlex
- W2043692200
- Cited by
- 462
- References
- 24
- FWCI
- 4.40
- Type
- journal article
- Language
- en
- Open access
- hybrid
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- Chen, Z.-Y., He, C.-Y., Ehrhardt, A., & Kay, M. A. (2003). Minicircle DNA vectors devoid of bacterial DNA result in persistent and high-level transgene expression in vivo. Molecular Therapy. https://doi.org/10.1016/s1525-0016(03)00168-0
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Contains information from the Semantic Scholar Academic Graph Datasets, made available under the ODC Attribution License (ODC-BY 1.0).
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