A new dual-vector approach to enhance recombinant adeno-associated virus-mediated gene expression through intermolecular cis activation
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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 [15]
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 [52]
Cites
Structural Analysis of Adeno-Associated Virus Transduction Circular Intermediates
Duan, D., Yan, Z., Yue, Y., & Engelhardt, J. F. (1999) · Virology · doi:10.1006/viro.1999.9821 · Cited by 100 · see citing works
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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
Grieger, J. C., & Samulski, R. J. (2005) · Journal of Virology · doi:10.1128/jvi.79.15.9933-9944.2005 · Cited by 419 · see citing works
Gene Therapy with Viral Vectors
Kootstra, N. A., & Verma, I. M. (2003) · Annual Review of Pharmacology and Toxicology · doi:10.1146/annurev.pharmtox.43.100901.140257 · Cited by 267 · see citing works
Yan, Z., Zak, R., Luxton, G. W. G., Ritchie, T. C., Bantel-Schaal, U., & Engelhardt, J. F. (2002) · Journal of Virology · doi:10.1128/jvi.76.5.2043-2053.2002 · Cited by 222 · see citing works
Self-citationExpressing Transgenes That Exceed the Packaging Capacity of Adeno-Associated Virus Capsids
Chamberlain, K., Riyad, J. M., & Weber, T. (2016) · Human Gene Therapy Methods · doi:10.1089/hgtb.2015.140 · Cited by 141 · see citing works
Haberman, R. P., McCown, T. J., & Samulski, R. J. (2000) · Journal of Virology · doi:10.1128/jvi.74.18.8732-8739.2000 · Cited by 139 · see citing works
AAV.Dysferlin Overlap Vectors Restore Function in Dysferlinopathy Animal Models
Sondergaard, P. C., Griffin, D. A., Pozsgai, E. R., Johnson, R. W., Grose, W. E., Heller, K. N., Shontz, K. M., Montgomery, C. L., Liu, J., Clark, K. R., et al. (2015) · Annals of Clinical and Translational Neurology · doi:10.1002/acn3.172 · Cited by 99 · see citing works
Grose, W. E., Clark, K. R., Griffin, D., Malik, V., Shontz, K. M., Montgomery, C. L., Lewis, S., Brown, R. H., Janssen, P. M. L., Mendell, J. R., et al. (2012) · PLoS ONE · doi:10.1371/journal.pone.0039233 · Cited by 76 · see citing works
Ghosh, A., Yue, Y., & Duan, D. (2006) · The Journal of Gene Medicine · doi:10.1002/jgm.835 · Cited by 69 · see citing works
Self-citationMarrone, L., Marchi, P. M., & Azzouz, M. (2022) · Expert Opinion on Biological Therapy · doi:10.1080/14712598.2022.2012148 · Cited by 56 · see citing works
Xu, Z., Yue, Y., Lai, Y., Ye, C., Qiu, J., Pintel, D. J., & Duan, D. (2004) · Human Gene Therapy · doi:10.1089/hum.2004.15.896 · Cited by 55 · see citing works
Self-citation
- Authors
- Duan, D., Yue, Y., Yan, Z., & Engelhardt, J. F.
- Affiliations
- University of Iowa (US)
- Corresponding author
- Engelhardt, John F.
- Venue
- Nature Medicine
- Published
- 2000-05
- Volume
- 6
- Issue
- 5
- Pages
- 595-598
- ISSN
- 1078-8956 (ISSN-L)
- 1546-170X
- OpenAlex
- W1582366477
- Cited by
- 200
- References
- 1
- Citations by year
- 2026: 5 · 2025: 10 · 2024: 5 · 2023: 3 · 2022: 4 · 2021: 7 · 2020: 5 · 2019: 5 · 2018: 3 · 2017: 4 · 2016: 4 · 2015: 3 · 2014: 1 · 2013: 7 · 2012: 6
- FWCI
- 11.71
- Type
- journal article
- Language
- en
- Open access
- closed
- Licence
- www.springer.com/tdm (tdm)
- Locations
- Nature Medicine (publishedVersion)
- PubMed (publishedVersion)
- Funders
- National Heart, Lung, and Blood Institute (R01HL058340, HL58430)
- National Institute of Diabetes and Digestive and Kidney Diseases (P30DK054759)
- 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
- recombinant adeno-associated virus, cis-acting enhancer, gene expression enhancement, adeno-associated virus vectors, gene therapy
- MeSH terms
- Dependovirus, Animals, Fibroblasts, Gene Expression Regulation, Genetic Vectors, Humans, Nucleic Acid Conformation, Recombinant Proteins, Gene Transfer Techniques, Muscle, Skeletal, Transgenes, Mice
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