The DOI matches its Crossref record; 6 of the 6 references it cites were found in a registry or an index.
Long-term pharmacologically regulated expression of erythropoietin in primates following AAV-mediated gene transfer
Abstract
Gene therapy is a potential route for the delivery of secreted therapeutic proteins, but pharmacologic control of expression will generally be required for optimal safety and efficacy. Previous attempts to achieve regulated expression in largeanimal models have been thwarted by transient expression or immune responses to regulatory proteins. We evaluated the ability of the dimerizer-regulated gene expression system to achieve controlled, long-term production of erythropoietin (Epo) following intramuscular administration of adeno-associated virus (AAV) vectors to 16 primates. All animals showed dose-responsive and completely reversible elevation of Epo and hematocrit in response to the dimerizer rapamycin, or analogs with reduced immunosuppressive activity, administered intravenously or orally. Animals that received optimized dual vectors showed persistent regulated expression for the duration of the study, with no apparent immune response to Epo or the regulatory proteins. Similar results were obtained with single vectors incorporating both the Epo and regulatory genes, including those packaged into serotype 1 AAV vectors to allow use of lower viral doses. For the longest-studied animal, regulated expression has persisted for more than 6 years and 26 induction cycles. These data indicate that one-time or infrequent gene transfer followed by dimerizer regulation is a promising approach for delivery of therapeutic proteins.
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Cites
AAV-mediated factor IX gene transfer to skeletal muscle in patients with severe hemophilia B
Manno, C. S., Chew, A. J., Hutchison, S., Larson, P. J., Herzog, R. W., Arruda, V. R., Tai, S. J., Ragni, M. V., Thompson, A., Ozelo, M., et al. (2003) · Blood · doi:10.1182/blood-2002-10-3296 · Cited by 749
Gene 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-citationChao, H., Liu, Y., Rabinowitz, J., Li, C., Samulski, R. J., & Walsh, C. E. (2000) · Molecular Therapy · doi:10.1006/mthe.2000.0219 · Cited by 366
Mount, J. D., Herzog, R. W., Tillson, D. M., Goodman, S. A., Robinson, N., McCleland, M. L., Bellinger, D., Nichols, T. C., Arruda, V. R., Lothrop, C. D., et al. (2002) · Blood · doi:10.1182/blood.v99.8.2670 · Cited by 339
Chirmule, N., Xiao, W., Truneh, A., Schnell, M. A., Hughes, J. V., Zoltick, P., & Wilson, J. M. (2000) · Journal of Virology · doi:10.1128/jvi.74.5.2420-2425.2000 · Cited by 198
Self-citationFavre, D., Blouin, V., Provost, N., Spisek, R., Porrot, F., Bohl, D., Marmé, F., Chérel, Y., Salvetti, A., Hurtrel, B., et al. (2002) · Journal of Virology · doi:10.1128/jvi.76.22.11605-11611.2002 · Cited by 150
Cited by
Viral vector platforms within the gene therapy landscape
Bulcha, J. T., Wang, Y., Ma, H., Tai, P. W. L., & Gao, G. (2021) · Signal Transduction and Targeted Therapy · doi:10.1038/s41392-021-00487-6 · Cited by 1,404
Self-citationAdeno-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
Self-citationChemically induced proximity in biology and medicine
Stanton, B. Z., Chory, E. J., & Crabtree, G. R. (2018) · Science · doi:10.1126/science.aao5902 · Cited by 475
Artificial viruses: a nanotechnological approach to gene delivery
Mastrobattista, E., van der Aa, M. A. E. M., Hennink, W. E., & Crommelin, D. J. A. (2006) · Nature Reviews Drug Discovery · doi:10.1038/nrd1960 · Cited by 346
Adeno-Associated Virus Vector Genomes Persist as Episomal Chromatin in Primate Muscle
Penaud-Budloo, M., Le Guiner, C., Nowrouzi, A., Toromanoff, A., Chérel, Y., Chenuaud, P., Schmidt, M., von Kalle, C., Rolling, F., Moullier, P., et al. (2008) · Journal of Virology · doi:10.1128/jvi.00649-08 · Cited by 317
Metadata
- Authors
- Rivera, V. M., Gao, G.-P., Grant, R. L., Schnell, M. A., Zoltick, P. W., Rozamus, L. W., Clackson, T., & Wilson, J. M.
- Venue
- Blood
- Publisher
- American Society of Hematology
- Published
- 2005-02-15
- Volume
- 105
- Issue
- 4
- Pages
- 1424-1430
- ISSN
- 0006-4971 (ISSN-L)
- 1528-0020
- OpenAlex
- W2054677438
- Cited by
- 276
- References
- 6
- FWCI
- 9.77
- Type
- journal article
- Language
- en
- Open access
- closed
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- Rivera, V. M., Gao, G.-P., Grant, R. L., Schnell, M. A., Zoltick, P. W., Rozamus, L. W., Clackson, T., & Wilson, J. M. (2005). Long-term pharmacologically regulated expression of erythropoietin in primates following AAV-mediated gene transfer. Blood. https://doi.org/10.1182/blood-2004-06-2501
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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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