The DOI matches its Crossref record; 33 of the 33 references it cites were found in a registry or an index.
Long-Term Restoration of Rod and Cone Vision by Single Dose rAAV-Mediated Gene Transfer to the Retina in a Canine Model of Childhood Blindness
Open accessAbstract
The short- and long-term effects of gene therapy using AAV-mediated RPE65 transfer to canine retinal pigment epithelium were investigated in dogs affected with disease caused by RPE65 deficiency. Results with AAV 2/2, 2/1, and 2/5 vector pseudotypes, human or canine RPE65 cDNA, and constitutive or tissue-specific promoters were similar. Subretinally administered vectors restored retinal function in 23 of 26 eyes, but intravitreal injections consistently did not. Photoreceptoral and postreceptoral function in both rod and cone systems improved with therapy. In dogs followed electroretinographically for 3 years, responses remained stable. Biochemical analysis of retinal retinoids indicates that mutant dogs have no detectable 11-cis-retinal, but markedly elevated retinyl esters. Subretinal AAV-RPE65 treatment resulted in detectable 11-cis-retinal expression, limited to treated areas. RPE65 protein expression was limited to retinal pigment epithelium of treated areas. Subretinal AAV-RPE65 vector is well tolerated and does not elicit high antibody levels to the vector or the protein in ocular fluids or serum. In long-term studies, wild-type cDNA is expressed only in target cells. Successful, stable restoration of rod and cone photoreceptor function in these dogs has important implications for treatment of human patients affected with Leber congenital amaurosis caused by RPE65 mutations.
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Cites
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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 · cited 13×
“This is also concordant with previous results demonstrating, by genomic PCR, that viral DNA persists only in the neural retina and RPE/ choroid of the injected quadrant [7].”
BackgroundMethodResultInfluentialSelf-citationRpe65 is necessary for production of 11-cis-vitamin A in the retinal visual cycle
Redmond., T. M., Yu, S., Lee, E., Bok, D., Hamasaki, D., Chen, N., Goletz, P., Ma, J.-X., Crouch, R. K., & Pfeifer, K. (1998) · Nature Genetics · doi:10.1038/3813 · Cited by 929
Production and purification of serotype 1, 2, and 5 recombinant adeno-associated viral vectors
Zolotukhin, S., Potter, M., Zolotukhin, I., Sakai, Y., Loiler, S., Fraites, T. J., Chiodo, V. A., Phillipsberg, T., Muzyczka, N., Hauswirth, W. W., et al. (2002) · Methods · doi:10.1016/s1046-2023(02)00220-7 · Cited by 565
Self-citationMcBee, J. K., Palczewski, K., Baehr, W., & Pepperberg, D. R. (2001) · Progress in Retinal and Eye Research · doi:10.1016/s1350-9462(01)00002-7 · Cited by 366 · cited 1×
“retinal pigment epithelium (RPE) with a key role in the cycling of retinoids [4–6].”
BackgroundSelf-citationHanein, S., Perrault, I., Gerber, S., Tanguy, G., Barbet, F., Ducroq, D., Calvas, P., Dollfus, H., Hamel, C., Lopponen, T., et al. (2004) · Human Mutation · doi:10.1002/humu.20010 · Cited by 350
Jacobson, S. G., Aleman, T. S., Cideciyan, A. V., Sumaroka, A., Schwartz, S. B., Windsor, E. A. M., Traboulsi, E. I., Heon, E., Pittler, S. J., Milam, A. H., et al. (2005) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.0500646102 · Cited by 274
Self-citationVan Hooser, J. P., Aleman, T. S., He, Y.-G., Cideciyan, A. V., Kuksa, V., Pittler, S. J., Stone, E. M., Jacobson, S. G., & Palczewski, K. (2000) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.150236297 · Cited by 252
Self-citationFunctional and structural recovery of the retina after gene therapy in the RPE65 null mutation dog
Narfstro¨m, K., Katz, M. L., Bragadottir, R., Seeliger, M., Boulanger, A., Redmond, T. M., Caro, L., Lai, C.-M., & Rakoczy, P. E. (2003) · Investigative Opthalmology & Visual Science · doi:10.1167/iovs.02-0595 · Cited by 252
Bennett, J., Maguire, A. M., Cideciyan, A. V., Schnell, M., Glover, E., Anand, V., Aleman, T. S., Chirmule, N., Gupta, A. R., Huang, Y., et al. (1999) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.96.17.9920 · Cited by 242
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Cited by
Hartong, D. T., Berson, E. L., & Dryja, T. P. (2006) · The Lancet · doi:10.1016/s0140-6736(06)69740-7 · Cited by 2,924
Safety and Efficacy of Gene Transfer for Leber's Congenital Amaurosis
Maguire, A. M., Simonelli, F., Pierce, E. A., Pugh, E. N., Mingozzi, F., Bennicelli, J., Banfi, S., Marshall, K. A., Testa, F., Surace, E. M., et al. (2008) · New England Journal of Medicine · doi:10.1056/nejmoa0802315 · Cited by 2,145
Self-citationRussell, S., Bennett, J., Wellman, J. A., Chung, D. C., Yu, Z.-F., Tillman, A., Wittes, J., Pappas, J., Elci, O., McCague, S., et al. (2017) · The Lancet · doi:10.1016/s0140-6736(17)31868-8 · Cited by 1,952
Self-citationHamel, C. (2006) · Orphanet Journal of Rare Diseases · doi:10.1186/1750-1172-1-40 · Cited by 962
Hauswirth, W. W., Aleman, T. S., Kaushal, S., Cideciyan, A. V., Schwartz, S. B., Wang, L., Conlon, T. J., Boye, S. L., Flotte, T. R., Byrne, B. J., et al. (2008) · Human Gene Therapy · doi:10.1089/hum.2008.107 · Cited by 954
Self-citation
Metadata
- Authors
- Acland, G. M., Aguirre, G. D., Bennett, J., Aleman, T. S., Cideciyan, A. V., Bennicelli, J., Dejneka, N. S., Pearce-Kelling, S. E., Maguire, A. M., Palczewski, K., Hauswirth, W. W., & Jacobson, S. G.
- Venue
- Molecular Therapy
- Publisher
- Elsevier BV
- Published
- 2005-12
- Volume
- 12
- Issue
- 6
- Pages
- 1072-1082
- ISSN
- 1525-0016 (ISSN-L)
- OpenAlex
- W1996875707
- Cited by
- 477
- References
- 33
- FWCI
- 13.25
- Type
- journal article
- Language
- en
- Open access
- hybrid
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- Acland, G. M., Aguirre, G. D., Bennett, J., Aleman, T. S., Cideciyan, A. V., Bennicelli, J., Dejneka, N. S., Pearce-Kelling, S. E., Maguire, A. M., Palczewski, K., Hauswirth, W. W., & Jacobson, S. G. (2005). Long-Term Restoration of Rod and Cone Vision by Single Dose rAAV-Mediated Gene Transfer to the Retina in a Canine Model of Childhood Blindness. Molecular Therapy. https://doi.org/10.1016/j.ymthe.2005.08.008
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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.
