ArticleThe journal of gene medicine2006
Viral serotype and the transgene sequence influence overlapping adeno-associated viral (AAV) vector-mediated gene transfer in skeletal muscle.
Article in The journal of gene medicine, 2006. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 38 papers.
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Who cites it
38 citing papers in PubMed, 66 citations in OpenAlex.
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- Double-strand break repair pathways differentially affect processing and transduction by dual AAV vectors.Nature communications · 2025Article
- Extracellular Vesicle-Encapsulated Adeno-Associated Viruses for Therapeutic Gene Delivery to the Heart.Circulation · 2023Article
- Screening for gene doping transgenes in horses via the use of massively parallel sequencing.Gene therapy · 2022Article
- Adeno-associated Virus (AAV) Dual Vector Strategies for Gene Therapy Encoding Large Transgenes.The Yale journal of biology and medicine · 2017Review
- Expressing Transgenes That Exceed the Packaging Capacity of Adeno-Associated Virus Capsids.Human gene therapy methods · 2016Review
- Gene therapy to rescue retinal degeneration caused by mutations in rhodopsin.Methods in molecular biology (Clifton, N.J.) · 2015Article
- A comparison of AAV strategies distinguishes overlapping vectors for efficient systemic delivery of the 6.2 kb Dysferlin coding sequence.Molecular therapy. Methods & clinical development · 2015Article
- Efficient production of dual recombinant adeno-associated viral vectors for factor VIII delivery.Human gene therapy methods · 2014Article
- Full-length dystrophin reconstitution with adeno-associated viral vectors.Human gene therapy · 2014Article
- The potential of adeno-associated viral vectors for gene delivery to muscle tissue.Expert opinion on drug delivery · 2014Review
- Effective delivery of large genes to the retina by dual AAV vectors.EMBO molecular medicine · 2014Article
- An AAV vector-mediated gene delivery approach facilitates reconstitution of functional human CD8+ T cells in mice.PloS one · 2014Article
- Dual AAV therapy ameliorates exercise-induced muscle injury and functional ischemia in murine models of Duchenne muscular dystrophy.Human molecular genetics · 2013Article
- Pseudotyped adeno-associated viral vectors for gene transfer in dermal fibroblasts: implications for wound-healing applications.The Journal of surgical research · 2013Article
- Retinal gene therapy with a large MYO7A cDNA using adeno-associated virus.Gene therapy · 2013Article
- Gene and cell-mediated therapies for muscular dystrophy.Muscle & nerve · 2013Review
- Gene therapy for retinal disease.Translational research : the journal of laboratory and clinical medicine · 2013Review
- A simplified immune suppression scheme leads to persistent micro-dystrophin expression in Duchenne muscular dystrophy dogs.Human gene therapy · 2012Article
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Authors and funding
3 authors at 1 institution in 1 country.
Funding
Abstract
backgroundThe overlapping approach was developed recently to expand the adeno-associated viral (AAV) packaging capacity. In this approach, a gene is split into two partially overlapping fragments and separately packaged into an upstream and a downstream vector, respectively. Transgene expression is achieved in co-infected cells after homologous recombination. Despite the promising proof-of-principle results in the lung, the efficiency has been very disappointing in skeletal muscle. Here we examined two potential rate-limiting factors including AAV serotype and the transgene sequence.
methodsTo study serotype effect, we delivered AAV-2, -5 and -6 overlapping vectors (5 x 10(8) vg particles of the upstream and the downstream vectors, respectively) and 5 x 10(8) vg particles of the intact gene vector to the tibialis anterior muscles of 7-week-old C57Bl/6 mice, respectively. To determine the effect of transgene sequence, we compared LacZ and alkaline phosphatase (AP) overlapping vectors. Transduction efficiency was quantified 6 weeks later by scoring the percentage of transgene-positive myofibers.
resultsAAV-2 overlapping vectors barely resulted in detectable transduction. Transduction efficiency was significantly improved in AAV-5 and AAV-6. The highest level was achieved in AAV-6 that reached 42% and 96% of that of the intact gene vector for the LacZ gene and the AP gene, respectively. Surprisingly, AAV-6 overlapping vector resulted in higher transduction than did AAV-2 and AAV-5 intact gene vectors.
conclusionsOur findings suggest that AAV serotype and the transgene sequence play critical roles in the overlapping approach. AAV-6 holds great promise for overlapping vector-mediated muscle gene therapy.
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