Dual Vector Expansion of the Recombinant AAV Packaging Capacity
Abstract
Adeno-associated virus (AAV) as a vector for gene therapy of cardiovascular diseases has received much recent attention (, , , , , ). This enthusiasm is based on the success of numerous proof of principal gene transfer experiments. Stable high-level expression has been achieved following a variety of delivery methods (direct cardiac muscle injection, coronary vascular or carotid artery infection) and in a wide range of animal models from rodents to pigs (, , , , , ). A side-by-side comparison between viral and nonviral vectors in rabbit heart suggests that rAAV is much more efficient than nonviral vectors such as naked or liposome-complexed DNA. At the same time, rAAV also displays the least inflammatory response in comparison with other viral vectors such as adenovirus and herpes simplex virus (). Regulated myocardial transgene expression has also been demonstrated with an AAV vector containing glucocorticoid response elements in rat heart ().
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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 [13]
Cites
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Trans-splicing vectors expand the utility of adeno-associated virus for gene therapy
Yan, Z., Zhang, Y., Duan, D., & Engelhardt, J. F. (2000) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.97.12.6716 · Cited by 305 · see citing works
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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
Ghosh, A., Yue, Y., & Duan, D. (2006) · The Journal of Gene Medicine · doi:10.1002/jgm.835 · Cited by 69 · see citing works
Self-citationLai, Y., Yue, Y., Liu, M., & Duan, D. (2006) · Human Gene Therapy · doi:10.1089/hum.2006.17.1036 · Cited by 37 · see citing works
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- Authors
- Duan, D., Yue, Y., & Engelhardt, J. F.
- Affiliations
- University of Missouri (US)
- University of Iowa (US)
- Venue
- Cardiac Cell and Gene Transfer
- Publisher
- Humana Press
- Published
- 2003-11-15
- Volume
- 219
- Pages
- 29-52
- OpenAlex
- W2415370792
- Cited by
- 26
- References
- 3
- Citations by year
- 2023: 1 · 2022: 1 · 2021: 1 · 2020: 1 · 2019: 1 · 2018: 1 · 2012: 1
- FWCI
- 6.66
- Type
- book chapter
- Language
- en
- Open access
- closed
- Locations
- Humana Press eBooks (publishedVersion)
- PubMed (publishedVersion)
- Funders
- National Institutes of Health
- National Heart, Lung, and Blood Institute (R01HL058340, 2R01 HL 58340)
- National Institute of Diabetes and Digestive and Kidney Diseases (P30 DK 54759, 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 › Neuroscience › Neurology › Barrier Structure and Function Studies
- Keywords
- adeno-associated virus, recombinant adeno-associated virus, gene therapy vectors, cardiovascular gene therapy, viral vectors, non-viral vectors, transgene expression regulation
- MeSH terms
- Dependovirus, Animals, Base Sequence, Cell Line, Genetic Vectors, Humans, Plasmids, Recombination, Genetic, Genetic Therapy, Gene Transfer Techniques
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