ArticleProceedings of the National Academy of Sciences of the United States of America2006
Enhancement of plasmid-mediated gene therapy for muscular dystrophy by directed plasmid integration.
Article in Proceedings of the National Academy of Sciences of the United States of America, 2006. 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, 105 citations in OpenAlex.
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- The current status of various preclinical therapeutic approaches for tendon repair.Annals of medicine · 2024Review
- Screening for gene doping transgenes in horses via the use of massively parallel sequencing.Gene therapy · 2022Article
- Basic Research on Tendon Repair: Strategies, Evaluation, and Development.Frontiers in medicine · 2021Review
- Plasmid-Mediated Gene Therapy in Mouse Models of Limb Girdle Muscular Dystrophy.Molecular therapy. Methods & clinical development · 2019Article
- Non-invasive monitoring of alternative splicing outcomes to identify candidate therapies for myotonic dystrophy type 1.Nature communications · 2018Article
- DNA-Mediated Gene Therapy in a Mouse Model of Limb Girdle Muscular Dystrophy 2B.Molecular therapy. Methods & clinical development · 2017Article
- Blastocyst Formation Rate and Transgene Expression are Associated with Gene Insertion into Safe and Non-Safe Harbors in the Cattle Genome.Scientific reports · 2017Article
- Biomaterial-Guided Gene Delivery for Musculoskeletal Tissue Repair.Tissue engineering. Part B, Reviews · 2017Review
- Programmable Genome Editing Tools and their Regulation for Efficient Genome Engineering.Computational and structural biotechnology journal · 2017Review
- Gene therapies that restore dystrophin expression for the treatment of Duchenne muscular dystrophy.Human genetics · 2016Review
- Clinical potential of ataluren in the treatment of Duchenne muscular dystrophy.Degenerative neurological and neuromuscular disease · 2016Review
- Long-term and efficient expression of human β-globin gene in a hematopoietic cell line using a new site-specific integrating non-viral system.Gene therapy · 2015Article
- Neuroprotective therapies in glaucoma: II. Genetic nanotechnology tools.Frontiers in neuroscience · 2015Review
- Intragenic integration in DLC1 sustains factor VIII expression in primary human cells without insertional oncogenicity.Gene therapy · 2014Article
- Hybrid lentivirus-phiC31-int-NLS vector allows site-specific recombination in murine and human cells but induces DNA damage.PloS one · 2014Article
- Emerging gene editing strategies for Duchenne muscular dystrophy targeting stem cells.Frontiers in physiology · 2014Review
- Expanding the scope of site-specific recombinases for genetic and metabolic engineering.Biotechnology and bioengineering · 2014Review
- Inorganic nanovectors for nucleic acid delivery.Drug delivery and translational research · 2013Article
- Gene and cell-mediated therapies for muscular dystrophy.Muscle & nerve · 2013Review
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
Plasmid-mediated gene therapy can restore dystrophin expression in skeletal muscle in the mdx mouse, a model of Duchenne muscular dystrophy. However, sufficient long-term expression and distribution of dystrophin remain a hurdle for translating this technology into a viable treatment for Duchenne muscular dystrophy. To improve plasmid-mediated gene therapy for muscle diseases, we studied the effects of targeted plasmid integration using a phage integrase (phiC31) that can mediate the integration of suitably modified plasmids into the mammalian genome. Using a luciferase expression plasmid, we monitored plasmid gene expression noninvasively in living mice by bioluminescence imaging. Coinjection of an integrase plasmid resulted in 5- to 10-fold higher levels of sustained luciferase expression. Likewise, plasmid-mediated dystrophin expression in mdx muscle was enhanced by integration. Using a combination of dystrophin and luciferase plasmids, we analyzed the functional benefit of dystrophin expression in the dystrophic muscle. The expression of dystrophin slowed the loss of luciferase expression associated with muscle degeneration, and that protection was enhanced by targeted integration of the dystrophin plasmid. In the presence of integrase, dystrophin expression was distributed along a much greater length of individual fibers, and this was associated with increased protection against degenerative changes. These data demonstrate the importance of both the level and distribution of dystrophin expression to achieve therapeutic efficacy, and that the efficacy can be enhanced by targeted plasmid integration.
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