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Tyrosine capsid-mutant AAV vectors for gene delivery to the canine retina from a subretinal or intravitreal approach
Open accessAbstract
Recombinant adeno-associated viruses are important vectors for retinal gene delivery. Currently utilized vectors have relatively slow onset, and for efficient transduction it is necessary to deliver treatment subretinally, with the potential for damage to the retina. Amino-acid substitutions in the viral capsid improve efficiency in rodent eyes by evading host responses. As dogs are important large animal models for human retinitis pigmentosa, we evaluated the speed and efficiency of retinal transduction using capsid-mutant vectors injected both subretinally and intravitreally. We evaluated AAV serotypes 2 and 8 with amino-acid substitutions of surface-exposed capsid tyrosine residues. The chicken beta-actin promoter was used to drive green fluorescent protein expression. Twelve normal adult beagles were injected; four dogs received intravitreal injections and eight dogs received subretinal injections. Capsid-mutant viruses tested included AAV2(quad Y-F) (intravitreal and subretinal) and self-complementary scAAV8(Y733F) (subretinal only). Contralateral control eyes received injections of scAAV5 (subretinal) or scAAV2 (intravitreal). Subretinally delivered vectors had a faster expression onset than intravitreally delivered vectors. Subretinally delivered scAAV8(Y733F) had a faster onset of expression than scAAV5. All subretinally injected vector types transduced the outer retina with high efficiency and the inner retina with moderate efficiency. Intravitreally delivered AAV2(quad Y-F) had a marginally higher efficiency of transduction of both outer retinal and inner retinal cells than scAAV2. Because of their rapid expression onset and efficient transduction, subretinally delivered capsid-mutant AAV8 vectors may increase the efficacy of gene therapy treatment for rapid photoreceptor degenerative diseases. With further refinement, capsid-mutant AAV2 vectors show promise for retinal gene delivery from an intravitreal approach.
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“Since surface-exposed residues on AAV capsids can be ubiquitinated through phosphorylation, mutations of these residues (e.g. tyrosine, lysine, serine) to reduce the likelihood of ubiquitination can generate novel capsids with reduced ubiquitination, antigen presentation, and activated cellular immunity (92 -99).”
From A versatile toolkit for overcoming AAV immunity (2022) · cites this paper as [96]
In the citing paper
“Subretinal gene delivery has been shown to be more effective and have a faster onset than intravitreal delivery in animal models [260,285,286].”
From Bruch’s Membrane: A Key Consideration with Complement-Based Therapies for Age-Related Macular Degeneration (2023) · cites this paper as [285]
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Rosenfeld, P. J., Brown, D. M., Heier, J. S., Boyer, D. S., Kaiser, P. K., Chung, C. Y., & Kim, R. Y. (2006) · New England Journal of Medicine · doi:10.1056/nejmoa054481 · Cited by 5,886
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
Effect of gene therapy on visual function in Leber’s congenital amaurosis
Bainbridge, J. W. B., Smith, A. J., Barker, S. S., Robbie, S., Henderson, R., Balaggan, K., Viswanathan, A., Holder, G. E., Stockman, A., Tyler, N., et al. (2008) · New England Journal of Medicine · doi:10.1056/nejmoa0802268 · Cited by 1,924
Self-citationGene 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-citation(1984) · PubMed · Cited by 1,200
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-citationBi, A., Cui, J., Ma, Y.-P., Olshevskaya, E. V., Pu, M., Dizhoor, A. M., & Pan, Z. (2006) · Neuron · doi:10.1016/j.neuron.2006.02.026 · Cited by 790
Dalkara, D., Byrne, L. C., Klimczak, R. R., Visel, M., Yin, L., Merigan, W. H., Flannery, J. G., & Schaffer, D. V. (2013) · Science Translational Medicine · doi:10.1126/scitranslmed.3005708 · Cited by 734
Jacobson, S. G. (2012) · Archives of Ophthalmology · doi:10.1001/archophthalmol.2011.298 · Cited by 639
Zhong, L., Li, B., Mah, C. S., Govindasamy, L., Agbandje-McKenna, M., Cooper, M., Herzog, R. W., Zolotukhin, I., Warrington, K. H., Weigel-Van Aken, K. A., et al. (2008) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.0802866105 · Cited by 558
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Molecular insights into PCARE-associated retinal disease
Serrano, J. C. C. (2018) · Radboud Repository (Radboud University)
Wei, Z., Liu, X., Li, T., Li, X., Zhou, Q., Wu, J., & Zhang, C. (2021) · Future Virology · doi:10.2217/fvl-2020-0096
AlGhosain, H. M., Nie, J., Liu, T., & Lee, J. (2024) · Research Square · doi:10.21203/rs.3.rs-3961400/v1
Suppressing microtubule detyrosination augments AAV2 endosomal escape and gene deliveryPreprint
Tripathi, S., Huda, S., Kar, J., Chandra, D., Giridhara, J. R., & Mohan, N. (2025) · bioRxiv (Cold Spring Harbor Laboratory) · doi:10.1101/2025.08.12.669862
Development of safe and efficient aav vectors for retinal gene therapy
Khabou, H. (2018) · theses.fr (ABES) · doi:10.70675/aa896663z9772z4d87z9bd1z247af1f0e3f1
Modification chimique d’AAV pour la thérapie génique
Leray, A. (2021) · theses.fr (ABES) · doi:10.70675/c33b1ba2z6c22z40e1z9a98z3ed26b698b7a
Beckwith‐Cohen, B., Sun, K., Occelli, L. M., Winkler, P. A., Somma, A. T., Montiani‐Ferreira, F., Marinho, L. F. L. P., Schall, P. Z., Parys, M., Yuzbasiyan‐Gurkan, V., et al. (2026) · Molecular Therapy Advances · doi:10.1016/j.omta.2026.201782
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Metadata
- Authors
- Mowat, F. M., Gornik, K. R., Dinculescu, A., Boye, S. L., Hauswirth, W. W., Petersen-Jones, S. M., & Bartoe, J. T.
- Affiliations
- Michigan State University (US)
- University of Florida Health (US)
- Venue
- Gene Therapy
- Published
- 2013-11-14
- Volume
- 21
- Issue
- 1
- Pages
- 96-105
- ISSN
- 0969-7128 (ISSN-L)
- 1476-5462
- PMC
- PMC3880610
- OpenAlex
- W2011980241
- Cited by
- 108
- References
- 64
- Citations by year
- 2026: 3 · 2025: 3 · 2024: 4 · 2023: 8 · 2022: 9 · 2021: 18 · 2020: 9 · 2019: 7 · 2018: 11 · 2017: 12 · 2016: 11 · 2015: 9 · 2014: 4
- FWCI
- 5.29
- Type
- journal article
- Language
- en
- Open access
- green
- www.ncbi.nlm.nih.gov
- Locations
- Gene Therapy (publishedVersion)
- PubMed (publishedVersion)
- PubMed Central (acceptedVersion)
- Funders
- National Eye Institute (P30EY021721, R24EY022023, EY-021721, R01EY017549)
- Topics
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Genetics › Virus-based gene therapy research
- Health Sciences › Medicine › Ophthalmology › Retinal Development and Disorders
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › RNA Interference and Gene Delivery
- Keywords
- adeno-associated virus vectors, retinal gene delivery, subretinal injection, intravitreal injection, canine retina, retinitis pigmentosa, AAV2, AAV8, self-complementary AAV, photoreceptor degeneration, beagle dogs
- MeSH terms
- Dependovirus, Animals, Capsid, Dogs, Female, Genetic Vectors, Humans, Male, Mutation, Recombinant Proteins, Retina, Transduction, Genetic, Tyrosine, Amino Acid Substitution, Viral Tropism, Injections, Intraocular
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