The DOI matches its Crossref record; 4 of the 4 references it cites were found in a registry or an index.
Purification of Recombinant Adeno-Associated Virus Vectors by Column Chromatography and Its Performance in Vivo
Abstract
Recombinant adeno-associated virus (AAV) holds much promise for human gene therapy. While evidence indicates that AAV mediates long-term gene transfer in several different tissues, difficulty in preparing and purifying this viral vector in large quantities remains a major obstacle for evaluating AAV vectors in clinical trials. The current method of purification, based on sedimentation through cesium chloride, is not scaleable and yields product of insufficient quality. In this article we report a new technique for purifying AAV, using a fully closed two-column chromatography system. Yields of AAV vectors purified by this method are high, potency is increased, and the purity of column-purified preparations is substantially improved. We previously reported a novel method to generate AAV based on an AAV Rep/Cap-containing cell line (B50) and an Ad-AAV hybrid virus, which is amenable to scale-up in bioreactors. By combining the new, fully scaleable purification process we report here with the B50/hybrid production method, it would be feasible to prepare AAV vectors to the scale and purity required for clinical and potential commercial applications.
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Cites
Recombinant adeno-associated virus for muscle directed gene therapy
Fisher, K. J., Jooss, K., Alston, J., Yang, Y., Haecker, S. E., High, K., Pathak, R., Raper, S. E., & Wilson, J. M. (1997) · Nature Medicine · doi:10.1038/nm0397-306 · Cited by 654
Self-citationGene therapy vectors based on adeno-associated virus type 1
Xiao, W., Chirmule, N., Berta, S. C., McCullough, B., Gao, G., & Wilson, J. M. (1999) · Journal of Virology · doi:10.1128/jvi.73.5.3994-4003.1999 · Cited by 541
Self-citationSamulski, R. J., Berns, K. I., Tan, M., & Muzyczka, N. (1982) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.79.6.2077 · Cited by 466
Cloning of infectious adeno-associated virus genomes in bacterial plasmids
Laughlin, C. A., Tratschin, J.-D., Coon, H., & Carter, B. J. (1983) · Gene · doi:10.1016/0378-1119(83)90217-2 · Cited by 266
Cited by
Novel adeno-associated viruses from rhesus monkeys as vectors for human gene therapy
Gao, G.-P., Alvira, M. R., Wang, L., Calcedo, R., Johnston, J., & Wilson, J. M. (2002) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.182412299 · Cited by 1,520
Self-citationWorldwide epidemiology of neutralizing antibodies to adeno-associated viruses
Calcedo, R., Vandenberghe, L. H., Gao, G., Lin, J., & Wilson, J. M. (2009) · The Journal of Infectious Diseases · doi:10.1086/595830 · Cited by 808
Self-citationCearley, C. N., & Wolfe, J. H. (2006) · Molecular Therapy · doi:10.1016/j.ymthe.2005.11.015 · Cited by 409
Allocca, M., Doria, M., Petrillo, M., Colella, P., Garcia-Hoyos, M., Gibbs, D., Kim, S. R., Maguire, A., Rex, T. S., Di Vicino, U., et al. (2008) · Journal of Clinical Investigation · doi:10.1172/jci34316 · Cited by 292
Manufacturing challenges and rational formulation development for AAV viral vectors
Srivastava, A., Mallela, K. M. G., Deorkar, N., & Brophy, G. (2021) · Journal of Pharmaceutical Sciences · doi:10.1016/j.xphs.2021.03.024 · Cited by 269
Metadata
- Authors
- Gao, G., Qu, G., Burnham, M. S., Huang, J., Chirmule, N., Joshi, B., Yu, Q.-C., Marsh, J. A., Conceicao, C. M., & Wilson, J. M.
- Venue
- Human Gene Therapy
- Publisher
- SAGE Publications
- Published
- 2000-10
- Volume
- 11
- Issue
- 15
- Pages
- 2079-2091
- ISSN
- 1043-0342 (ISSN-L)
- 1557-7422
- OpenAlex
- W2081694771
- Cited by
- 174
- References
- 4
- FWCI
- 4.82
- Type
- journal article
- Language
- en
- Open access
- closed
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- CoChat. (2026). Citation check: Purification of Recombinant Adeno-Associated Virus Vectors by Column Chromatography and Its Performance in Vivo. https://app.cochat.ai/papers/10.1089/104303400750001390
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- Gao, G., Qu, G., Burnham, M. S., Huang, J., Chirmule, N., Joshi, B., Yu, Q.-C., Marsh, J. A., Conceicao, C. M., & Wilson, J. M. (2000). Purification of Recombinant Adeno-Associated Virus Vectors by Column Chromatography and Its Performance in Vivo. Human Gene Therapy. https://doi.org/10.1089/104303400750001390
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Contains information from the Semantic Scholar Academic Graph Datasets, made available under the ODC Attribution License (ODC-BY 1.0).
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