The DOI matches its Crossref record; 60 of the 60 references it cites were found in a registry or an index.
Adeno-Associated Virus Vector Genomes Persist as Episomal Chromatin in Primate Muscle
Open accessAbstract
Recombinant adeno-associated virus (rAAV) vectors are capable of mediating long-term gene expression following administration to skeletal muscle. In rodent muscle, the vector genomes persist in the nucleus in concatemeric episomal forms. Here, we demonstrate with nonhuman primates that rAAV vectors integrate inefficiently into the chromosomes of myocytes and reside predominantly as episomal monomeric and concatemeric circles. The episomal rAAV genomes assimilate into chromatin with a typical nucleosomal pattern. The persistence of the vector genomes and gene expression for years in quiescent tissues suggests that a bona fide chromatin structure is important for episomal maintenance and transgene expression. These findings were obtained from primate muscles transduced with rAAV1 and rAAV8 vectors for up to 22 months after intramuscular delivery of 5 × 10 12 viral genomes/kg. Because of this unique context, our data, which provide important insight into in situ vector biology, are highly relevant from a clinical standpoint.
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Cites
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Cited by
Adeno-associated virus as a delivery vector for gene therapy of human diseases
Wang, J.-H., Gessler, D. J., Zhan, W., Gallagher, T. L., & Gao, G. (2024) · Signal Transduction and Targeted Therapy · doi:10.1038/s41392-024-01780-w · Cited by 904
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Metadata
- Authors
- Penaud-Budloo, M., Le Guiner, C., Nowrouzi, A., Toromanoff, A., Chérel, Y., Chenuaud, P., Schmidt, M., von Kalle, C., Rolling, F., Moullier, P., & Snyder, R. O.
- Venue
- Journal of Virology
- Publisher
- American Society for Microbiology
- Published
- 2008-08-15
- Volume
- 82
- Issue
- 16
- Pages
- 7875-7885
- ISSN
- 0022-538X (ISSN-L)
- 1098-5514
- OpenAlex
- W2072123447
- Cited by
- 317
- References
- 60
- FWCI
- 4.92
- Type
- journal article
- Language
- en
- Open access
- green
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- Penaud-Budloo, M., Le Guiner, C., Nowrouzi, A., Toromanoff, A., Chérel, Y., Chenuaud, P., Schmidt, M., von Kalle, C., Rolling, F., Moullier, P., & Snyder, R. O. (2008). Adeno-Associated Virus Vector Genomes Persist as Episomal Chromatin in Primate Muscle. Journal of Virology. https://doi.org/10.1128/jvi.00649-08
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Contains information from the Semantic Scholar Academic Graph Datasets, made available under the ODC Attribution License (ODC-BY 1.0).
Semantic Scholar’s data is described in: Kinney, R., et al. (2023). The Semantic Scholar Open Data Platform. arXiv:2301.10140.
