The DOI matches its Crossref record; 4 of the 4 references it cites were found in a registry or an index.
Biology of AAV Serotype Vectors in Liver-Directed Gene Transfer to Nonhuman Primates
Open accessAbstract
Vectors based on adeno-associated viruses (AAVs) show promise for the treatment of genetic diseases. This study evaluates the biology of AAV-mediated gene transfer to liver in nonhuman primates (NHPs) using vectors based on AAV serotypes 2, 7, and 8. Transgenes encoding self-proteins were selected to minimize the confounding development of transgene-specific immune responses. These included the beta subunit of choriogonadotropic hormone (bCG) and erythropoietin (Epo), both derived from cDNAs from rhesus macaques. Experiments were performed with bCG in rhesus macaques and Epo in cynomolgus macaques. We demonstrated the previously untested hypothesis that preexisting immunity to a natural infection does substantially diminish the efficacy of gene transfer with a vector derived from an endogenous virus. Route of vector administration clearly has an impact on the development of immune responses to self-antigens. In general, efficiency of gene transfer to liver with AAV7 and 8 vectors was higher than what was achieved with AAV2, although a variety of host factors may influence this important parameter, such as preexisting immunity, gender, and transgene immunity.
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Gene therapy vectors based on adeno-associated virus type 1
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Self-citationSamulski, R. J., Srivastava, A., Berns, K. I., & Muzyczka, N. (1983) · Cell · doi:10.1016/0092-8674(83)90342-2 · Cited by 297
Genetic Fate of Recombinant Adeno-Associated Virus Vector Genomes in Muscle
Schnepp, B. C., Clark, K. R., Klemanski, D. L., Pacak, C. A., & Johnson, P. R. (2003) · Journal of Virology · doi:10.1128/jvi.77.6.3495-3504.2003 · Cited by 262
Yan, Z., Zak, R., Zhang, Y., & Engelhardt, J. F. (2005) · Journal of Virology · doi:10.1128/jvi.79.1.364-379.2005 · Cited by 107
Cited by
Emerging issues in AAV-mediated in vivo gene therapy
Colella, P., Ronzitti, G., & Mingozzi, F. (2018) · Molecular Therapy - Methods & Clinical Development · doi:10.1016/j.omtm.2017.11.007 · Cited by 881
Engineering adeno-associated viruses for clinical gene therapy
Kotterman, M. A., & Schaffer, D. V. (2014) · Nature Reviews Genetics · doi:10.1038/nrg3742 · Cited by 761
Grimm, D., Lee, J. S., Wang, L., Desai, T., Akache, B., Storm, T. A., & Kay, M. A. (2008) · Journal of Virology · doi:10.1128/jvi.00254-08 · Cited by 696
Self-citationRobust cellular reprogramming occurs spontaneously during liver regeneration
Yanger, K., Zong, Y., Maggs, L. R., Shapira, S. N., Maddipati, R., Aiello, N. M., Thung, S. N., Wells, R. G., Greenbaum, L. E., & Stanger, B. Z. (2013) · Genes & Development · doi:10.1101/gad.207803.112 · Cited by 484
Jiang, H., Couto, L. B., Patarroyo-White, S., Liu, T., Nagy, D., Vargas, J. A., Zhou, S., Scallan, C. D., Sommer, J., Vijay, S., et al. (2006) · Blood · doi:10.1182/blood-2006-04-017913 · Cited by 330
Metadata
- Authors
- Gao, G., Lu, Y., Calcedo, R., Grant, R. L., Bell, P., Wang, L., Figueredo, J., Lock, M., & Wilson, J. M.
- Venue
- Molecular Therapy
- Publisher
- Elsevier BV
- Published
- 2006-01
- Volume
- 13
- Issue
- 1
- Pages
- 77-87
- ISSN
- 1525-0016 (ISSN-L)
- OpenAlex
- W2091177028
- Cited by
- 170
- References
- 4
- FWCI
- 5.21
- Type
- journal article
- Language
- en
- Open access
- hybrid
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- Gao, G., Lu, Y., Calcedo, R., Grant, R. L., Bell, P., Wang, L., Figueredo, J., Lock, M., & Wilson, J. M. (2006). Biology of AAV Serotype Vectors in Liver-Directed Gene Transfer to Nonhuman Primates. Molecular Therapy. https://doi.org/10.1016/j.ymthe.2005.08.017
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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.
