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Trans-splicing vectors expand the utility of adeno-associated virus for gene therapy
Open accessAbstract
Adeno-associated viral (AAV) vectors have demonstrated considerable promise for gene therapy of inherited diseases. However, with a packaging size of <5 kb, applications have been limited to relatively small disease genes. Based on the finding that AAV genomes undergo intermolecular circular concatamerization after transduction in muscle, we have developed a paradigm to increase the size of delivered transgenes with this vector through trans-splicing between two independent vectors coadministered to the same tissue. When two vectors encoding either the 5′ or 3′ portions of the erythropoietin genomic locus were used, functional erythropoietin protein was expressed in muscle subsequent to the formation of intermolecular circular concatamers in a head-to-tail orientation through trans-splicing between these two independent vector genomes. These findings will allow for the application of AAV technologies to a wider variety of diseases for which therapeutic transgenes exceed the packaging limitation of present AAV vectors.
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“All of these methods require multiple vectors, and the efficiency of some of these methods is inherently limited by the efficiencies of intron processing or homologous recombination.”
From Effect of genome size on AAV vector packaging (2010) · cites this paper as [5]
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“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 [52]
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“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 [53]
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Metadata
- Authors
- Yan, Z., Zhang, Y., Duan, D., & Engelhardt, J. F.
- Affiliations
- University of Iowa (US)
- Venue
- Proceedings of the National Academy of Sciences
- Publisher
- National Academy of Sciences
- Published
- 2000-06-06
- Volume
- 97
- Issue
- 12
- Pages
- 6716-6721
- ISSN
- 0027-8424 (ISSN-L)
- 1091-6490
- PMC
- PMC18714
- OpenAlex
- W2082328650
- Cited by
- 305
- References
- 20
- Citations by year
- 2026: 3 · 2025: 9 · 2024: 9 · 2023: 10 · 2022: 5 · 2021: 12 · 2020: 12 · 2019: 14 · 2018: 9 · 2017: 10 · 2016: 7 · 2015: 11 · 2014: 9 · 2013: 14 · 2012: 7
- FWCI
- 13.07
- Type
- journal article
- Language
- en
- Open access
- green
- www.ncbi.nlm.nih.gov
- Locations
- Proceedings of the National Academy of Sciences (publishedVersion)
- Europe PMC (PubMed Central) (publishedVersion)
- PubMed Central (publishedVersion)
- PubMed (publishedVersion)
- Funders
- National Heart, Lung, and Blood Institute (R01HL058340, R01 HL58340)
- 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
- Health Sciences › Medicine › Genetics (clinical) › Neurogenetic and Muscular Disorders Research
- Keywords
- adeno-associated virus vectors, trans-splicing, erythropoietin, gene therapy
- MeSH terms
- Dependovirus, Anemia, Animals, Erythropoietin, Genetic Vectors, Kidney Failure, Chronic, Mice, Inbred C57BL, Muscles, Genetic Therapy, Mice
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