Viral serotype and the transgene sequence influence overlapping adeno‐associated viral (AAV) vector‐mediated gene transfer in skeletal muscle
Open accessAbstract
Background: The 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. Methods: To study serotype effect, we delivered AAV‐2, ‐5 and ‐6 overlapping vectors (5 × 10 8 vg particles of the upstream and the downstream vectors, respectively) and 5 × 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. Results: AAV‐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. Conclusions: Our 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. Copyright © 2005 John Wiley & Sons, Ltd.
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The 872 bp AP fragment is highly recombinogenic in the overlapping vector system.
“The choice of the 872 bp AP fragment was based on earlier work that showed that this AP fragment is highly recombinogenic in the overlapping vector system. 66”
From Expressing Transgenes That Exceed the Packaging Capacity of Adeno-Associated Virus Capsids (2016) · cites this paper as [66]
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- Authors
- Ghosh, A., Yue, Y., & Duan, D.
- Affiliations
- University of Missouri (US)
- Corresponding author
- Duan, Dongsheng
- Venue
- The Journal of Gene Medicine
- Publisher
- Wiley
- Published
- 2005-12-29
- Volume
- 8
- Issue
- 3
- Pages
- 298-305
- ISSN
- 1099-498X (ISSN-L)
- 1521-2254
- PMC
- PMC2581716
- OpenAlex
- W2084129972
- Cited by
- 69
- References
- 47
- Citations by year
- 2026: 1 · 2025: 4 · 2024: 1 · 2023: 2 · 2021: 2 · 2020: 2 · 2019: 3 · 2018: 2 · 2017: 1 · 2016: 1 · 2015: 5 · 2014: 7 · 2013: 6 · 2012: 2
- FWCI
- 2.70
- Type
- journal article
- Language
- en
- Open access
- green
- www.ncbi.nlm.nih.gov
- Locations
- The Journal of Gene Medicine (publishedVersion)
- PubMed (publishedVersion)
- PubMed Central (acceptedVersion)
- Funders
- National Institutes of Health
- National Institute of Arthritis and Musculoskeletal and Skin Diseases (AR49419, R01AR049419)
- Topics
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Genetics › Virus-based gene therapy research
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › RNA Interference and Gene Delivery
- Health Sciences › Medicine › Genetics › Neurogenetic and Muscular Disorders Research
- Keywords
- adeno-associated virus, AAV2, skeletal muscle gene transfer, homologous recombination, transduction efficiency, tibialis anterior, alkaline phosphatase, lacZ reporter
- MeSH terms
- Adenoviridae, Alkaline Phosphatase, Animals, Genes, Overlapping, Genetic Vectors, Lac Operon, Male, Mice, Inbred C57BL, Plasmids, Serotyping, Transduction, Genetic, Gene Transfer Techniques, Muscle, Skeletal, Transgenes, Muscular Dystrophy, Duchenne, Mice, In Vitro Techniques
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