The DOI matches its Crossref record; 4 of the 4 references it cites were found in a registry or an index.
Stable transgene expression in rod photoreceptors after recombinant adeno-associated virus-mediated gene transfer to monkey retina
Open accessAbstract
Recombinant adeno-associated virus (rAAV) is a promising vector for therapy of retinal degenerative diseases. We evaluated the efficiency, cellular specificity, and safety of retinal cell transduction in nonhuman primates after subretinal delivery of an rAAV carrying a cDNA encoding green fluorescent protein (EGFP), rAAV.CMV. EGFP . The treatment results in efficient and stable EGFP expression lasting >1 year. Transgene expression in the neural retina is limited exclusively to rod photoreceptors. There is neither electroretinographic nor histologic evidence of photoreceptor toxicity. Despite significant serum antibody responses to the vector, subretinal readministration results in additional transduction events. The findings further characterize the retinal cell tropism of rAAV. They also support the development of studies aimed ultimately at treating inherited retinal degeneration by using rAAV-mediated gene therapy.
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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-citationPhotoreceptor cell rescue in retinal degeneration (rd) mice by in vivo gene therapy
Bennett, J., Tanabe, T., Sun, D., Zeng, Y., Kjeldbye, H., Gouras, P., & Maguire, A. M. (1996) · Nature Medicine · doi:10.1038/nm0696-649 · Cited by 316
Self-citationStargardt's ABCR is localized to the disc membrane of retinal rod outer segments
Sun, H., & Nathans, J. (1997) · Nature Genetics · doi:10.1038/ng0997-15 · Cited by 251
Persistent transgene product in retina, optic nerve and brain after intraocular injection of rAAV
Dudus, L., Anand, V., Acland, G. M., Chen, S.-J., Wilson, J. M., Fisher, K. J., Maguire, A. M., & Bennett, J. (1999) · Vision Research · doi:10.1016/s0042-6989(98)00308-3 · Cited by 160
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Cited by
Gene therapy restores vision in a canine model of childhood blindness
Acland, G. M., Aguirre, G. D., Ray, J., Zhang, Q., Aleman, T. S., Cideciyan, A. V., Pearce-Kelling, S. E., Anand, V., Zeng, Y., Maguire, A. M., et al. (2001) · Nature Genetics · doi:10.1038/ng0501-92 · Cited by 1,202
Self-citationAAV2 Gene Therapy Readministration in Three Adults with Congenital Blindness
Bennett, J., Ashtari, M., Wellman, J., Marshall, K. A., Cyckowski, L. L., Chung, D. C., McCague, S., Pierce, E. A., Chen, Y., Bennicelli, J. L., et al. (2012) · Science Translational Medicine · doi:10.1126/scitranslmed.3002865 · Cited by 395
Self-citationAxons and synaptic boutons are highly dynamic in adult visual cortex
Stettler, D. D., Yamahachi, H., Li, W., Denk, W., & Gilbert, C. D. (2006) · Neuron · doi:10.1016/j.neuron.2006.02.018 · Cited by 256
Dosage thresholds for AAV2 and AAV8 photoreceptor gene therapy in monkey
Vandenberghe, L. H., Bell, P., Maguire, A. M., Cearley, C. N., Xiao, R., Calcedo, R., Wang, L., Castle, M. J., Maguire, A. C., Grant, R., et al. (2011) · Science Translational Medicine · doi:10.1126/scitranslmed.3002103 · Cited by 242
Self-citationBasic Biology of Adeno-Associated Virus (AAV) Vectors Used in Gene Therapy
Balakrishnan, B., & Jayandharan, G. (2014) · Current Gene Therapy · doi:10.2174/1566523214666140302193709 · Cited by 225
Metadata
- Authors
- Bennett, J., Maguire, A. M., Cideciyan, A. V., Schnell, M., Glover, E., Anand, V., Aleman, T. S., Chirmule, N., Gupta, A. R., Huang, Y., Gao, G.-P., Nyberg, W. C., Tazelaar, J., Hughes, J., Wilson, J. M., & Jacobson, S. G.
- Venue
- Proceedings of the National Academy of Sciences
- Published
- 1999-08-17
- Volume
- 96
- Issue
- 17
- Pages
- 9920-9925
- ISSN
- 0027-8424 (ISSN-L)
- 1091-6490
- OpenAlex
- W2064199560
- Cited by
- 242
- References
- 4
- FWCI
- 5.65
- Type
- journal article
- Language
- en
- Open access
- green
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- The paper
- Bennett, J., Maguire, A. M., Cideciyan, A. V., Schnell, M., Glover, E., Anand, V., Aleman, T. S., Chirmule, N., Gupta, A. R., Huang, Y., Gao, G.-P., Nyberg, W. C., Tazelaar, J., Hughes, J., Wilson, J. M., & Jacobson, S. G. (1999). Stable transgene expression in rod photoreceptors after recombinant adeno-associated virus-mediated gene transfer to monkey retina. Proceedings of the National Academy of Sciences. https://doi.org/10.1073/pnas.96.17.9920
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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.
