Overcoming adeno-associated virus vector size limitation through viral DNA heterodimerization
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Split vector systems exploiting head-to-tail concatamerization have been developed to circumvent the small packaging capacity of AAV genomes.
“Recently, split vector systems, exploiting head-to-tail concatamerization formation, have been developed to circumvent the small packaging capacity of AAV genomes (13-16, 41, 52).”
From Packaging Capacity of Adeno-Associated Virus Serotypes: Impact of Larger Genomes on Infectivity and Postentry Steps (2005) · cites this paper as [41]
The trans-splicing method to expand the size of ORFs that can be successfully expressed using AAV vectors was originally developed in the laboratory of John Engelhardt and has since been utilized in several studies with varying success.
“The trans-splicing method to expand the size of ORFs that can be successfully expressed using AAV vectors was originally developed in the laboratory of John Engelhardt 52,53 and has since been utilized in several studies with varying success (e.g., refs. 53-57 ).”
From Expressing Transgenes That Exceed the Packaging Capacity of Adeno-Associated Virus Capsids (2016) · cites this paper as [57]
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“3 a ) orientations, and has also demonstrated many variant ITR structures (data not shown), which might be the result of ITR recombination and/or ligatio”
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Progress and problems with the use of viral vectors for gene therapy
Thomas, C. E., Ehrhardt, A., & Kay, M. A. (2003) · Nature Reviews Genetics · doi:10.1038/nrg1066 · Cited by 2,501 · see citing works
Adeno-associated virus vector as a platform for gene therapy delivery
Wang, D., Tai, P. W. L., & Gao, G. (2019) · Nature Reviews Drug Discovery · doi:10.1038/s41573-019-0012-9 · Cited by 2,444 · see citing works
Viral vector platforms within the gene therapy landscape
Bulcha, J. T., Wang, Y., Ma, H., Tai, P. W. L., & Gao, G. (2021) · Signal Transduction and Targeted Therapy · doi:10.1038/s41392-021-00487-6 · Cited by 1,404 · see citing works
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Gene Therapy with Viral Vectors
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Expressing Transgenes That Exceed the Packaging Capacity of Adeno-Associated Virus Capsids
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- Authors
- Sun, L., Li, J., & Xiao, X.
- Affiliations
- University of Pittsburgh (US)
- Corresponding author
- Xiao, Xiao
- ORCID
- Xiao, Xiao
- Venue
- Nature Medicine
- Published
- 2000-05
- Volume
- 6
- Issue
- 5
- Pages
- 599-602
- ISSN
- 1078-8956 (ISSN-L)
- 1546-170X
- OpenAlex
- W1527888432
- Cited by
- 267
- References
- 2
- Citations by year
- 2026: 6 · 2025: 8 · 2024: 11 · 2023: 5 · 2022: 4 · 2021: 11 · 2020: 13 · 2019: 8 · 2018: 3 · 2017: 5 · 2016: 7 · 2015: 5 · 2014: 2 · 2013: 4 · 2012: 6
- FWCI
- 13.66
- Type
- journal article
- Language
- en
- Open access
- closed
- Licence
- www.springer.com/tdm (tdm)
- Locations
- Nature Medicine (publishedVersion)
- PubMed (publishedVersion)
- Funders
- National Institutes of Health (1r21dk055966-01)
- National Institute of Diabetes and Digestive and Kidney Diseases (R21 DK55966)
- National Institute of Arthritis and Musculoskeletal and Skin Diseases (AR45967, R01AR045967, P01AR045925, AR45925)
- 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
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › CRISPR and Genetic Engineering
- Keywords
- adeno-associated virus vectors, gene therapy vectors, viral genome size
- MeSH terms
- Dependovirus, Animals, Genetic Vectors, Humans, Lac Operon, Molecular Weight, Recombinant Proteins, Gene Transfer Techniques, Transgenes, Dimerization, Mice
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Registry, retraction, Cites and statistics results are computed, not judged, with one exception: a printed reference that no index lists is read once by a model before it counts as a citation error. Claims are extracted by a model and kept only when their quote appears verbatim in the text; each cited source is then checked, with a model’s help, for whether it says what the claim relies on. Those verdicts can be wrong — read the claim’s quote and the cited source before relying on one.
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