The DOI matches its Crossref record; 4 of the 4 references it cites were found in a registry or an index.
Highly Purified Recombinant Adeno-Associated Virus Vectors Are Biologically Active and Free of Detectable Helper and Wild-Type Viruses
Abstract
Gene transfer vectors based on the replication-defective human parvovirus, adeno-associated virus type 2 (AAV-2), are viable candidates for in vivo and ex vivo human use. However, widespread testing of AAV vectors has been limited by difficulties in generating pure, high-titer vector stocks that are fully characterized. To address these issues, we have developed a single-step purification scheme using heparin affinity chromatography. Recovery from the crude lysate starting material exceeds 70%, and the end product rAAV vector is highly purified and appears to be free of adenovirus and cellular contaminates. Importantly, purified vectors retain predicted in vivo biologic activity. Concurrently, we have developed simple and rapid approaches for vector quantification using real-time PCR. These new methods, combined with the use of stable producer cell lines for rAAV production, make the commercial production of rAAV vectors for human use truly viable and pragmatic.
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Cites
Ferrari, F. K., Samulski, T., Shenk, T., & Samulski, R. J. (1996) · Journal of Virology · doi:10.1128/jvi.70.5.3227-3234.1996 · Cited by 761
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
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
Recombinant Adeno-Associated Viral Vectors Mediate Long-Term Transgene Expression in Muscle
Clark, K. R., Sferra, T. J., & Johnson, P. R. (1997) · Human Gene Therapy · doi:10.1089/hum.1997.8.6-659 · Cited by 153
Self-citation
Cited by
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
The Endocannabinoid System Controls Key Epileptogenic Circuits in the Hippocampus
Monory, K., Massa, F., Egertová, M., Eder, M., Blaudzun, H., Westenbroek, R., Kelsch, W., Jacob, W., Marsch, R., Ekker, M., et al. (2006) · Neuron · doi:10.1016/j.neuron.2006.07.006 · Cited by 706
Infectious entry pathway of adeno-associated virus and adeno-associated virus vectors
Bartlett, J. S., Wilcher, R., & Samulski, R. J. (2000) · Journal of Virology · doi:10.1128/jvi.74.6.2777-2785.2000 · Cited by 532
Differential activation of innate immune responses by adenovirus and adeno-associated virus vectors
Zaiss, A.-K., Liu, Q., Bowen, G. P., Wong, N. C. W., Bartlett, J. S., & Muruve, D. A. (2002) · Journal of Virology · doi:10.1128/jvi.76.9.4580-4590.2002 · Cited by 394
Opie, S. R., Warrington, Agbandje-McKenna, M., Zolotukhin, S., & Muzyczka, N. (2003) · Journal of Virology · doi:10.1128/jvi.77.12.6995-7006.2003 · Cited by 364
Metadata
- Authors
- Clark, K. R., Liu, X., Mcgrath, J. P., & Johnson, P. R.
- Venue
- Human Gene Therapy
- Publisher
- SAGE Publications
- Published
- 1999-04-10
- Volume
- 10
- Issue
- 6
- Pages
- 1031-1039
- ISSN
- 1043-0342 (ISSN-L)
- 1557-7422
- OpenAlex
- W2180590412
- Cited by
- 305
- References
- 4
- FWCI
- 12.83
- Type
- journal article
- Language
- en
- Open access
- closed
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- Clark, K. R., Liu, X., Mcgrath, J. P., & Johnson, P. R. (1999). Highly Purified Recombinant Adeno-Associated Virus Vectors Are Biologically Active and Free of Detectable Helper and Wild-Type Viruses. Human Gene Therapy. https://doi.org/10.1089/10430349950018427
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Bibliographic data: Semantic Scholar, OpenAlex, Crossref.
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.
