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.
The claims and statistics of this paper haven’t been checked yet.
Run the check — free, no account needed. It reads the open-access text and checks the sources it cites.
Integrity signals
Registry
Cites
Statistics
Claims the paper makes
The claims and statistics of this paper haven’t been checked yet.
Run the check — free, no account needed. It reads the open-access text and checks the sources it cites.
Claims other papers make about this one
Each is checked against this paper’s own text: does it say what the citing paper says it does?
Surface-exposed residues on AAV capsids can be ubiquitinated through phosphorylation, and mutations of these residues to reduce the likelihood of ubiquitination can generate novel capsids with reduced ubiquitination, antigen presentation, and activated cellular immunity.
“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]
Subretinal gene delivery has been shown to be more effective and have a faster onset than intravitreal delivery in animal models.
“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]
Self-check
Percentages, sums and reported differences re-computed from the paper’s own numbers.
The self-check needs the paper’s full text.
Cites
Ranibizumab for Neovascular Age-Related Macular Degeneration
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 · see citing works
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 · see citing works
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 · see citing works
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 · see citing works
Self-citation(1984) · PubMed · Cited by 1,200 · see citing works
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 · see citing works
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 774 · see citing works
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 · see citing works
Jacobson, S. G. (2012) · Archives of Ophthalmology · doi:10.1001/archophthalmol.2011.298 · Cited by 639 · see citing works
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 · see citing works
Simonelli, F., Maguire, A. M., Testa, F., Pierce, E. A., Mingozzi, F., Bennicelli, J. L., Rossi, S., Marshall, K. A., Banfi, S., Surace, E. M., et al. (2009) · Molecular Therapy · doi:10.1038/mt.2009.277 · Cited by 553 · see citing works
Acland, G. M., Aguirre, G. K., Bennett, J., Alemán, T. S., Cideciyan, A. V., Bennicelli, J. L., Dejneka, N. S., Pearce-Kelling, S. E., Maguire, A. M., Palczewski, K., et al. (2005) · Molecular Therapy · doi:10.1016/j.ymthe.2005.08.008 · Cited by 478 · see citing works
Self-citationCideciyan, A. V., Jacobson, S. G., Beltran, W. A., Sumaroka, A., Świder, M., Iwabe, S., Román, A. J., Olivares, M. B., Schwartz, S., Komáromy, A. M., et al. (2013) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.1218933110 · Cited by 441 · see citing works
Self-citationHigh-efficiency transduction of the mouse retina by tyrosine-mutant AAV serotype vectors
Petrs-Silva, H., Dinculescu, A., Li, Q., Min, S.-H., Chiodo, V., Pang, J.-J., Zhong, L., Zolotukhin, S., Srivastava, A., Lewin, A. S., et al. (2009) · Molecular Therapy · doi:10.1038/mt.2008.269 · Cited by 398 · see citing works
Self-citationApoptotic photoreceptor degeneration in experimental retinal detachment.
Cook, B. E., Lewis, G. P., Fisher, S. K., & Adler, R. (1995) · PubMed · Cited by 367 · see citing works
Inner limiting membrane barriers to aav-mediated retinal transduction from the vitreous
Dalkara, D., Kolstad, K. D., Caporale, N., Visel, M., Klimczak, R. R., Schaffer, D. V., & Flannery, J. G. (2009) · Molecular Therapy · doi:10.1038/mt.2009.181 · Cited by 357 · see citing works
Cideciyan, A. V., Hauswirth, W. W., Aleman, T. S., Kaushal, S., Schwartz, S. B., Boye, S. L., Windsor, E. A. M., Conlon, T. J., Sumaroka, A., Pang, J.-J., et al. (2009) · Human Gene Therapy · doi:10.1089/hum.2009.086 · Cited by 312 · see citing works
Self-citationRestoration of visual function in retinal degeneration mice by ectopic expression of melanopsin
Lin, B., Koizumi, A., Tanaka, N., Panda, S., & Masland, R. H. (2008) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.0806114105 · Cited by 302 · see citing works
Doroudchi, M. M., Greenberg, K. P., Liu, J., Silka, K. A., Boyden, E. S., Lockridge, J. A., Arman, A. C., Ramesh, J., Boye, S. L., Boye, S. L., et al. (2011) · Molecular Therapy · doi:10.1038/mt.2011.69 · Cited by 274 · see citing works
Self-citationNovel properties of tyrosine-mutant AAV2 vectors in the mouse retina
Petrs-Silva, H., Dinculescu, A., Li, Q., Deng, W.-T., Pang, J.-J., Min, S.-H., Chiodo, V., Neeley, A. W., Govindasamy, L., Bennett, A., et al. (2011) · Molecular Therapy · doi:10.1038/mt.2010.234 · Cited by 267 · see citing works
Self-citationGene therapy rescues cone function in congenital achromatopsia
Komáromy, A. M., Alexander, J. J., Rowlan, J. S., Garcia, M. M., Chiodo, V. A., Kaya, A., Tanaka, J. C., Acland, G. M., Hauswirth, W. W., & Aguirre, G. D. (2010) · Human Molecular Genetics · doi:10.1093/hmg/ddq136 · Cited by 264 · see citing works
Self-citationA comprehensive review of retinal gene therapy
Boye, S. E., Boye, S. L., Lewin, A. S., & Hauswirth, W. W. (2013) · Molecular Therapy · doi:10.1038/mt.2012.280 · Cited by 262 · see citing works
Self-citationBeltran, W. A., Cideciyan, A. V., Lewin, A. S., Iwabe, S., Khanna, H., Sumaroka, A., Chiodo, V. A., Fajardo, D. S., Román, A. J., Deng, W.-T., et al. (2012) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.1118847109 · Cited by 260 · see citing works
Self-citationFisher, S. K., & Lewis, G. P. (2003) · Vision Research · doi:10.1016/s0042-6989(02)00680-6 · Cited by 215 · see citing works
Novel AAV serotypes for improved ocular gene transfer
Lebherz, C., Maguire, A. M., Tang, W., Bennett, J., & Wilson, J. M. (2008) · The Journal of Gene Medicine · doi:10.1002/jgm.1126 · Cited by 207 · see citing works
Meur, G. L., Stieger, K., Smith, A. J., Weber, M., Deschamps, J., Nivard, D., Mendes-Madeira, A., Provost, N., Péréon, Y., Chérel, Y., et al. (2006) · Gene Therapy · doi:10.1038/sj.gt.3302861 · Cited by 207 · see citing works
Targeting photoreceptors via intravitreal delivery using novel, capsid-mutated AAV vectors
Kay, C. N., Ryals, R. C., Aslanidi, G. V., Min, S. H., Ruan, Q., Sun, J., Dyka, F. M., Kasuga, D., Ayala, A. E., Van Vliet, K., et al. (2013) · PLoS ONE · doi:10.1371/journal.pone.0062097 · Cited by 168 · see citing works
Self-citationMartino, A. T., Basner-Tschakarjan, E., Markusic, D. M., Finn, J. D., Hinderer, C., Zhou, S., Ostrov, D. A., Srivastava, A., Ertl, H. C. J., Terhorst, C., et al. (2013) · Blood · doi:10.1182/blood-2012-10-460733 · Cited by 162 · see citing works
Tomita, H., Sugano, E., Yawo, H., Ishizuka, T., Isago, H., Narikawa, S., Kügler, S., & Tamai, M. (2007) · Investigative Ophthalmology & Visual Science · doi:10.1167/iovs.06-1501 · Cited by 156 · see citing works
Pang, J., Dai, X., Boye, S. L., Barone, I., Boye, S. L., Mao, S., Everhart, D., Dinculescu, A., Liu, L., Umino, Y., et al. (2010) · Molecular Therapy · doi:10.1038/mt.2010.273 · Cited by 148 · see citing works
Self-citationLi, Z., Zhao, W., Wu, J., Li, B., Zolotukhin, S., Lakshmanan Govindasamy, A., Agbandje‐McKenna, M., & Srivastava, A. (2007) · Molecular Therapy · doi:10.1038/sj.mt.6300170 · Cited by 146 · see citing works
Natkunarajah, M., Trittibach, P., McIntosh, J., Duran, Y., Barker, S. E., Smith, A. J., Nathwani, A. C., & Ali, R. R. (2008) · Gene Therapy · doi:10.1038/sj.gt.3303074 · Cited by 138 · see citing works
Zhang, Y., Ivanova, E., Bi, A., & Pan, Z. (2009) · Journal of Neuroscience · doi:10.1523/jneurosci.0184-09.2009 · Cited by 132 · see citing works
Petersen‐Jones, S. M., Entz, D. D., & Sargan, D. R. (1999) · PubMed · Cited by 123 · see citing works
Self-citationSchmitz-Valckenberg, S., Fleckenstein, M., Göbel, A. P., Hohman, T. C., & Holz, F. G. (2010) · Investigative Ophthalmology & Visual Science · doi:10.1167/iovs.10-5619 · Cited by 106 · see citing works
Aslanidi, G., Rivers, A. E., Ortiz, L., Song, L., Ling, C., Lakshmanan Govindasamy, A., Vliet, K. V., Tan, M., Agbandje‐McKenna, M., & Srivastava, A. (2013) · PLoS ONE · doi:10.1371/journal.pone.0059142 · Cited by 96 · see citing works
Stieger, K., Schroeder, J., Provost, N., Mendes-Madeira, A., Belbellaa, B., Meur, G. L., Weber, M., Deschamps, J.-Y., Lorenz, B., Moullier, P., et al. (2009) · Molecular Therapy · doi:10.1038/mt.2008.283 · Cited by 88 · see citing works
Retinal Thickness Normative Data in Wild-Type Mice Using Customized Miniature SD-OCT
Ferguson, L. R., Dominguez, J. M., Balaiya, S., Grover, S., & Chalam, K. V. (2013) · PLoS ONE · doi:10.1371/journal.pone.0067265 · Cited by 84 · see citing works
Current mutation discovery approaches in Retinitis Pigmentosa
Anasagasti, A., Irigoyen, C., Barandika, O., de Munaín, A. L., & Ruiz‐Ederra, J. (2012) · Vision Research · doi:10.1016/j.visres.2012.09.012 · Cited by 70 · see citing works
Li, A., Zhu, X., Brown, B., & Craft, C. M. (2003) · Investigative Ophthalmology & Visual Science · doi:10.1167/iovs.02-0434 · Cited by 66 · see citing works
Restoration of Vision in the pde6β-deficient Dog, a Large Animal Model of Rod-cone Dystrophy
Petit, L., Lhériteau, E., Weber, M., Meur, G. L., Deschamps, J., Provost, N., Mendes-Madeira, A., Libeau, L., Guihal, C., Colle, M.-A., et al. (2012) · Molecular Therapy · doi:10.1038/mt.2012.134 · Cited by 65 · see citing works
Direct Comparison of Administration Routes for AAV8-mediated Ocular Gene Therapy
Igarashi, T., Miyake, K., Asakawa, N., Miyake, N., Shimada, T., & Takahashi, H. (2013) · Current Eye Research · doi:10.3109/02713683.2013.779720 · Cited by 64 · see citing works
Boye, S. L., Conlon, T. J., Erger, K. E., Ryals, R. C., Neeley, A. W., Cossette, T. L., Pang, J., Dyka, F. M., Hauswirth, W. W., & Boye, S. L. (2011) · Investigative Ophthalmology & Visual Science · doi:10.1167/iovs.11-7867 · Cited by 63 · see citing works
Self-citationJacobson, S. G., Cideciyan, A. V., Alemán, T. S., Sumaroka, A., Román, A. J., Świder, M., Schwartz, S., Banin, E., & Stone, E. M. (2010) · Investigative Ophthalmology & Visual Science · doi:10.1167/iovs.10-6127 · Cited by 63 · see citing works
Changes in adeno-associated virus-mediated gene delivery in retinal degeneration
Kolstad, K. D., Dalkara, D., Guerin, K., Visel, M., Hoffmann, N., Schaffer, D. V., & Flannery, J. G. (2010) · Human Gene Therapy · doi:10.1089/hum.2009.194 · Cited by 63 · see citing works
Englander, M., Chen, T. C., Paschalis, E. I., Miller, J. W., & Kim, I. K. (2013) · British Journal of Ophthalmology · doi:10.1136/bjophthalmol-2012-302435 · Cited by 61 · see citing works
Tuntivanich, N., Pittler, S. J., Fischer, A. J., Omar, G., Kiupel, M., Weber, A. J., Yao, S., Steibel, J. P., Khan, N. W., & Petersen‐Jones, S. M. (2009) · Investigative Ophthalmology & Visual Science · doi:10.1167/iovs.08-2562 · Cited by 60 · see citing works
Self-citationSarra, G., Stephens, C., Schlichtenbrede, F. C., Bainbridge, J. W., Thrasher, A. J., Luthert, P. J., & Ali, R. R. (2002) · Vision Research · doi:10.1016/s0042-6989(01)00230-9 · Cited by 58 · see citing works
Tyrosine-Mutant AAV8 Delivery of Human MERTK Provides Long-Term Retinal Preservation in RCS Rats
Deng, W., Dinculescu, A., Li, Q., Boye, S. L., Li, J., Gorbatyuk, M. S., Pang, J., Chiodo, V. A., Matthes, M. T., Yasumura, D., et al. (2012) · Investigative Ophthalmology & Visual Science · doi:10.1167/iovs.11-8831 · Cited by 55 · see citing works
Self-citationGearhart, P. M. (2010) · Archives of Ophthalmology · doi:10.1001/archophthalmol.2010.210 · Cited by 52 · see citing works
Pseudotyped AAV vectors for constitutive and regulated gene expression in the eye
Auricchio, A. (2003) · Vision Research · doi:10.1016/s0042-6989(02)00676-4 · Cited by 50 · see citing works
PORES IN THE INTERNAL LIMITING MEMBRANE OF THE HUMAN RETINA
Wolter, J. R. (1964) · Acta Ophthalmologica · doi:10.1111/j.1755-3768.1964.tb03664.x · Cited by 49 · see citing works
Hernandez‐Merino, E., Kecova, H., Jacobson, S. J., Hamouche, K. N., Nzokwe, R. N., & Grozdanić, S. D. (2011) · Veterinary Ophthalmology · doi:10.1111/j.1463-5224.2011.00887.x · Cited by 48 · see citing works
Ku, C. A., Chiodo, V. A., Boye, S. L., Goldberg, A. F. X., Li, T., Hauswirth, W. W., & Ramamurthy, V. (2011) · Human Molecular Genetics · doi:10.1093/hmg/ddr391 · Cited by 46 · see citing works
Self-citationTesta, F., Surace, E. M., Rossi, S., Marrocco, E., Gargiulo, A., Iorio, V. D., Ziviello, C., Nesti, A., Fecarotta, S., Bacci, M. L., et al. (2011) · Investigative Ophthalmology & Visual Science · doi:10.1167/iovs.10-6543 · Cited by 44 · see citing works
Dinculescu, A., Estreicher, J., Zenteno, J. C., Alemán, T. S., Schwartz, S., Huang, W., Román, A. J., Sumaroka, A., Li, Q., Deng, W., et al. (2011) · Human Gene Therapy · doi:10.1089/hum.2011.169 · Cited by 43 · see citing works
Self-citationPetersen‐Jones, S. M., Bartoe, J. T., Fischer, A. J., Scott, M. A., Boye, S. L., Chiodo, V. A., & Hauswirth, W. W. (2009) · PubMed Central · Cited by 38 · see citing works
Self-citationResidual Electroretinograms in Young Leber Congenital Amaurosis Patients with Mutations of AIPL1
Pennesi, M. E., Stover, N. B., Stone, E. M., Chiang, P.-W., & Weleber, R. G. (2011) · Investigative Ophthalmology & Visual Science · doi:10.1167/iovs.11-8298 · Cited by 35 · see citing works
Structure of the Human Vitreoretinal Border Region
Heegaard, S. (2010) · Ophthalmologica · doi:10.1159/000310458 · Cited by 35 · see citing works
Fisher, S. K., Stone, J., Rex, T. S., Linberg, K. A., & Lewis, G. P. (2001) · Progress in brain research · doi:10.1016/s0079-6123(01)31053-1 · Cited by 31 · see citing works
Bruewer, A. R., Mowat, F. M., Bartoe, J. T., Boye, S. L., Hauswirth, W. W., & Petersen‐Jones, S. M. (2013) · PLoS ONE · doi:10.1371/journal.pone.0060218 · Cited by 29 · see citing works
Self-citationMowat, F. M., Bartoe, J. T., Bruewer, A. R., Dinculescu, A., Boye, S. L., Hauswirth, W. W., & Petersen‐Jones, S. M. (2012) · Investigative Ophthalmology & Visual Science · Cited by 7 · see citing works
Self-citationGarcía‐Sánchez, G. A., Gil‐Carrasco, F., Román, J. J., Brooks, D. E., Álvarez‐Clau, A., Hosgood, G. L., Iwabe, S., & Moreno-Méndoza, N. (2007) · Veterinary Ophthalmology · doi:10.1111/j.1463-5224.2007.00563.x · Cited by 6 · see citing works
RPE65 Gene Therapy Promotes Survival Of S-cones In The RPE65-deficient Dog
Mowat, F. M., Bartoe, J. T., Bruewer, A. R., Smith, A. J., Bainbridge, J. W., Ali, R. R., & Petersen‐Jones, S. M. (2011) · Investigative Ophthalmology & Visual Science · Cited by 1 · see citing works
Self-citation
Flagged first, then by citations
Cited by
Sorted by citations
Progresses towards safe and efficient gene therapy vectors
Chira, S., Jackson, C. S., Oprea, I. I., Öztürk, F., Pepper, M. S., Diaconu, I., Braicu, C., Ráduly, L., Călin, G. A., & Berindan‐Neagoe, I. (2015) · Oncotarget · doi:10.18632/oncotarget.5169 · Cited by 219 · see citing works
A robust activity marking system for exploring active neuronal ensembles
Sørensen, A. T., Cooper, Y. A., Baratta, M. V., Weng, F., Zhang, Y., Ramamoorthi, K., Fropf, R., LaVerriere, E., Xue, J., Young, A., et al. (2016) · eLife · doi:10.7554/elife.13918 · Cited by 189 · see citing works
Vector platforms for gene therapy of inherited retinopathies
Trapani, I., Puppo, A., & Auricchio, A. (2014) · Progress in Retinal and Eye Research · doi:10.1016/j.preteyeres.2014.08.001 · Cited by 184 · see citing works
Wassmer, S. J., Carvalho, L. S., György, B., Vandenberghe, L. H., & Maguire, C. A. (2017) · Scientific Reports · doi:10.1038/srep45329 · Cited by 164 · see citing works
Evaluation of Dose and Safety of AAV7m8 and AAV8BP2 in the Non-Human Primate Retina
Ramachandran, P. S., Lee, V., Wei, Z., Song, J. Y., Casal, G., Cronin, T., Willett, K., Huckfeldt, R., Morgan, J. I. W., Aleman, T. S., et al. (2017) · Human Gene Therapy · doi:10.1089/hum.2016.111 · Cited by 147 · see citing works
AAV8-vectored suprachoroidal gene transfer produces widespread ocular transgene expression
Ding, K., Shen, J., Hafiz, Z., Hackett, S. F., Silva, R. L. E., Khan, M., Lorenc, V. E., Chen, D., Chadha, R., Zhang, M., et al. (2019) · Journal of Clinical Investigation · doi:10.1172/jci129085 · Cited by 141 · see citing works
Novel AAV capsids for intravitreal gene therapy of photoreceptor disorders
Pavlou, M., Schön, C., Occelli, L. M., Rossi, A., Meumann, N., Boyd, R. F., Bartoe, J. T., Siedlecki, J., Gerhardt, M. J., Babutzka, S., et al. (2021) · EMBO Molecular Medicine · doi:10.15252/emmm.202013392 · Cited by 135 · see citing works
Self-citationIn vitro and ex vivo models to study drug delivery barriers in the posterior segment of the eye
Peynshaert, K., Devoldere, J., Smedt, S. C. D., & Remaut, K. (2017) · Advanced Drug Delivery Reviews · doi:10.1016/j.addr.2017.09.007 · Cited by 132 · see citing works
Seeing the light after 25 Years of retinal gene therapy
Trapani, I., & Auricchio, A. (2018) · Trends in Molecular Medicine · doi:10.1016/j.molmed.2018.06.006 · Cited by 124 · see citing works
Gene therapy for inherited retinal diseases: Progress and possibilities
Hu, M. L., Edwards, T. L., O’Hare, F., Hickey, D. G., Wang, J.-H., Liu, Z., & Ayton, L. N. (2021) · Clinical and Experimental Optometry · doi:10.1080/08164622.2021.1880863 · Cited by 118 · see citing works
Slijkerman, R. W. N., Song, F., Astuti, G., Huynen, M. A., van Wijk, E., Stieger, K., & Collin, R. W. J. (2015) · Progress in Retinal and Eye Research · doi:10.1016/j.preteyeres.2015.04.004 · Cited by 114 · see citing works
Peynshaert, K., Devoldere, J., Minnaert, A., Smedt, S. C. D., & Remaut, K. (2019) · Current Eye Research · doi:10.1080/02713683.2019.1565890 · Cited by 92 · see citing works
Treatment of ocular disorders by gene therapy
Solinís, M. Á., del Pozo-Rodríguez, A., Apaolaza, P. S., & Rodríguez-Gascón, A. (2015) · European Journal of Pharmaceutics and Biopharmaceutics · doi:10.1016/j.ejpb.2014.12.022 · Cited by 88 · see citing works
Bush, R. A., Zeng, Y., Colosi, P., Kjellstrom, S., Hiriyanna, S., Vijayasarathy, C., Santos, M., Li, J., Wu, Z., & Sieving, P. A. (2016) · Human Gene Therapy · doi:10.1089/hum.2015.142 · Cited by 86 · see citing works
Suprachoroidal delivery of viral and nonviral gene therapy for retinal diseases
Kansara, V., Muya, L., Wan, C.-R., & Ciulla, T. A. (2020) · Journal of Ocular Pharmacology and Therapeutics · doi:10.1089/jop.2019.0126 · Cited by 84 · see citing works
Advances in gene therapy for diseases of the eye
Petit, L., Khanna, H., & Punzo, C. (2016) · Human Gene Therapy · doi:10.1089/hum.2016.040 · Cited by 82 · see citing works
Highly Efficient Delivery of Adeno-Associated Viral Vectors to the Primate Retina
Boye, S. L., Alexander, J. J., Witherspoon, C. D., Boye, S. L., Peterson, J. J., Clark, M. E., Sandefer, K. J., Girkin, C. A., Hauswirth, W. W., & Gamlin, P. D. (2016) · Human Gene Therapy · doi:10.1089/hum.2016.085 · Cited by 78 · see citing works
Self-citationGonzalez-Cordero, A., Goh, D., Kruczek, K., Naeem, A., Fernando, M., kleine Holthaus, S.-M., Takaaki, M., Blackford, S. J. I., Kloc, M., Agundez, L., et al. (2018) · Human Gene Therapy · doi:10.1089/hum.2018.027 · Cited by 77 · see citing works
Khabou, H., Desrosiers, M., Winckler, C., Fouquet, S., Auregan, G., Bemelmans, A., Sahel, J., & Dalkara, D. (2016) · Biotechnology and Bioengineering · doi:10.1002/bit.26031 · Cited by 71 · see citing works
Retinal gene delivery by adeno-associated virus (AAV) vectors: Strategies and applications
Schön, C., Biel, M., & Michalakis, S. (2015) · European Journal of Pharmaceutics and Biopharmaceutics · doi:10.1016/j.ejpb.2015.01.009 · Cited by 68 · see citing works
Assessment of Adeno-Associated Virus Serotype Tropism in Human Retinal Explants
Wiley, L. A., Burnight, E. R., Kaalberg, E. E., Jiao, C., Riker, M. J., Halder, J. A., Luse, M. A., Han, I. C., Russell, S. R., Sohn, E. H., et al. (2017) · Human Gene Therapy · doi:10.1089/hum.2017.179 · Cited by 67 · see citing works
The future of retinal gene therapy: evolving from subretinal to intravitreal vector delivery
Ross, M., & Ofri, R. (2021) · Neural Regeneration Research · doi:10.4103/1673-5374.306063 · Cited by 67 · see citing works
“…not differ from that of WT AAV, mostly inner retinal transduction Dias et al. (2019) Dog Moderate photoreceptor transduction following IVT injection Mowat et al. (2014); Boyd et al. (2016) Sheep AAV quad (Y-F+T-V) injected IVT to sheep does not result in photoreceptor transduction Ross et al.…”
BackgroundInfluentialDevoldere, J., Peynshaert, K., Dewitte, H., Vanhove, C., Groef, L. D., Moons, L., Özcan, S. Y., Dalkara, D., Smedt, S. C. D., & Remaut, K. (2019) · Journal of Controlled Release · doi:10.1016/j.jconrel.2019.06.042 · Cited by 66 · see citing works
Zhang, K. Y., & Johnson, T. V. (2021) · Experimental Eye Research · doi:10.1016/j.exer.2021.108545 · Cited by 63 · see citing works
Ciulla, T. A., Hussain, R. M., Berrocal, A. M., & Nagiel, A. (2020) · Expert Opinion on Biological Therapy · doi:10.1080/14712598.2020.1740676 · Cited by 59 · see citing works
Peynshaert, K., Devoldere, J., Forster, V., Picaud, S. A., Vanhove, C., Smedt, S. C. D., & Remaut, K. (2017) · Drug Delivery · doi:10.1080/10717544.2017.1375578 · Cited by 58 · see citing works
A versatile toolkit for overcoming AAV immunity
Li, X., Wei, X., Lin, J., & Ou, L. (2022) · Frontiers in Immunology · doi:10.3389/fimmu.2022.991832 · Cited by 57 · see citing works
Gene Therapy in a Large Animal Model of PDE6A-Retinitis Pigmentosa
Mowat, F. M., Occelli, L. M., Bartoe, J. T., Gervais, K. J., Bruewer, A. R., Querubin, J. R., Dinculescu, A., Boye, S. L., Hauswirth, W. W., & Petersen‐Jones, S. M. (2017) · Frontiers in Neuroscience · doi:10.3389/fnins.2017.00342 · Cited by 54 · see citing works
Self-citationMutations in the Kinesin-2 Motor KIF3B Cause an Autosomal-Dominant Ciliopathy
Cogné, B., Latypova, X., Senaratne, L. D. S., Martin, L., Koboldt, D. C., Kellaris, G., Fievet, L., Meur, G. L., Caldari, D., Debray, D., et al. (2020) · The American Journal of Human Genetics · doi:10.1016/j.ajhg.2020.04.005 · Cited by 49 · see citing works
Self-citationThe Future Looks Brighter After 25 Years of Retinal Gene Therapy
Auricchio, A., Smith, A. J., & Ali, R. R. (2017) · Human Gene Therapy · doi:10.1089/hum.2017.164 · Cited by 53 · see citing works
Has retinal gene therapy come of age? From bench to bedside and back to bench
Trapani, I., & Auricchio, A. (2019) · Human Molecular Genetics · doi:10.1093/hmg/ddz130 · Cited by 51 · see citing works
Gene Therapy of Inherited Retinal Degenerations: Prospects and Challenges
Trapani, I., Banfi, S., Simonelli, F., Surace, E. M., & Auricchio, A. (2015) · Human Gene Therapy · doi:10.1089/hum.2015.030 · Cited by 46 · see citing works
Delivering AAV to the Central Nervous and Sensory Systems
Peters, C. W., Maguire, C. A., & Hanlon, K. S. (2021) · Trends in Pharmacological Sciences · doi:10.1016/j.tips.2021.03.004 · Cited by 44 · see citing works
Holthaus, S.-M. K., Ribeiro, J., Abelleira, L. E., Pearson, R. A., Durán, Y., Georgiadis, A., Sampson, R. D., Rizzi, M., Hoke, J., Maswood, R. N., et al. (2018) · Molecular Therapy · doi:10.1016/j.ymthe.2018.02.027 · Cited by 45 · see citing works
Hammadi, S., Tzoumas, N., Ferrara, M., Meschede, I. P., Lo, K., Harris, C., Lako, M., & Steel, D. H. (2023) · Journal of Clinical Medicine · doi:10.3390/jcm12082870 · Cited by 44 · see citing works
Mukherjee, S., Kuroiwa, M., Oakden, W., Paul, B. T., Noman, A., Chen, J., Lin, V., Dimitrijevic, A., Stanisz, G. J., & Le, T. (2021) · Molecular Therapy · doi:10.1016/j.ymthe.2021.07.013 · Cited by 42 · see citing works
The concept of gene therapy for glaucoma: the dream that has not come true yet
Sulak, R., Liu, X., & Smedowski, A. (2024) · Neural Regeneration Research · doi:10.4103/1673-5374.375319 · Cited by 40 · see citing works
Hashida, N., & Nishida, K. (2023) · Advanced Drug Delivery Reviews · doi:10.1016/j.addr.2023.114870 · Cited by 37 · see citing works
Holthaus, S.-M. K., Aristorena, M., Maswood, R. N., Semenyuk, O., Hoke, J., Hare, A., Smith, A. J., Mole, S., & Ali, R. R. (2020) · Human Gene Therapy · doi:10.1089/hum.2020.038 · Cited by 38 · see citing works
Photoreceptor-targeted gene delivery using intravitreally administered AAV vectors in dogs
Boyd, R. F., Sledge, D. G., Boye, S. L., Boye, S. E., Hauswirth, W. W., Komáromy, A. M., Petersen‐Jones, S. M., & Bartoe, J. T. (2015) · Gene Therapy · doi:10.1038/gt.2015.96 · Cited by 38 · see citing works
Self-citationBoyd, R. F., Boye, S. L., Conlon, T. J., Erger, K. E., Sledge, D. G., Langohr, I. M., Hauswirth, W. W., Komáromy, A. M., Boye, S. E., Petersen-Jones, S. M., et al. (2016) · Gene Therapy · doi:10.1038/gt.2016.31 · Cited by 37 · see citing works
Self-citationChen, M., Maeng, K., Nawab, A., Francois, R. A., Bray, J. K., Reinhard, M. K., Boye, S. L., Hauswirth, W. W., Kaye, F. J., Aslanidi, G., et al. (2017) · Human Gene Therapy Methods · doi:10.1089/hgtb.2016.089 · Cited by 35 · see citing works
Self-citationSanjurjo-Soriano, C., & Kalatzis, V. (2018) · Neural Plasticity · doi:10.1155/2018/5056279 · Cited by 35 · see citing works
Novel Adeno-associated Viruses Derived From Pig Tissues Transduce Most Major Organs in Mice
Bello, A., Chand, A., Aviles, J., Soule, G., Auricchio, A., & Kobinger, G. P. (2014) · Scientific Reports · doi:10.1038/srep06644 · Cited by 35 · see citing works
Occelli, L. M., Schön, C., Seeliger, M. W., Biel, M., Michalakis, S., & Petersen‐Jones, S. M. (2017) · Human Gene Therapy · doi:10.1089/hum.2017.155 · Cited by 35 · see citing works
Self-citationA novel retinal ganglion cell promoter for utility in AAV vectors
Hanlon, K. S., Chadderton, N., Palfi, A., Blanco Fernandez, A., Humphries, P., Kenna, P. F., Millington-Ward, S., & Farrar, G. J. (2017) · Frontiers in Neuroscience · doi:10.3389/fnins.2017.00521 · Cited by 34 · see citing works
Tshilenge, K., Ameline, B., Weber, M., Mendes-Madeira, A., Nédellec, S., Biget, M., Provost, N., Libeau, L., Blouin, V., Deschamps, J., et al. (2016) · Human Gene Therapy Methods · doi:10.1089/hgtb.2016.034 · Cited by 34 · see citing works
Devoldere, J., Wels, M., Peynshaert, K., Dewitte, H., Smedt, S. C. D., & Remaut, K. (2019) · European Journal of Pharmaceutics and Biopharmaceutics · doi:10.1016/j.ejpb.2019.05.023 · Cited by 30 · see citing works
Crx Rdy Cat: A Large Animal Model for CRX -Associated Leber Congenital Amaurosis
Occelli, L. M., Tran, N. M., Narfström, K. L., Chen, S., & Petersen‐Jones, S. M. (2016) · Investigative Ophthalmology & Visual Science · doi:10.1167/iovs.16-19444 · Cited by 29 · see citing works
Self-citationGene therapy for inherited retinal degenerations: initial successes and future challenges
Gupta, P. R., & Huckfeldt, R. M. (2017) · Journal of Neural Engineering · doi:10.1088/1741-2552/aa7a27 · Cited by 29 · see citing works
Tyrosine triple mutated AAV2-BDNF gene therapy in a rat model of transient IOP elevation.
Igarashi, T., Miyake, K., Kobayashi, M., Kameya, S., Fujimoto, C., Nakamoto, K., Takahashi, H., Igarashi, T., Miyake, N., Iijima, O., et al. (2016) · Europe PMC (PubMed Central) · Cited by 28 · see citing works
Long-Term Effects of Retinal Gene Therapy in Childhood Blindness
Wright, A. F. (2015) · New England Journal of Medicine · doi:10.1056/nejme1503419 · Cited by 28 · see citing works
Garoon, R. B., & Stout, J. T. (2016) · Current Opinion in Ophthalmology · doi:10.1097/icu.0000000000000256 · Cited by 25 · see citing works
X-Linked RetinoschisisPreprint
Ku, C. A., Wei, L. W., & Sieving, P. A. (2023) · Cold Spring Harbor Perspectives in Medicine · doi:10.1101/cshperspect.a041288 · Cited by 24 · see citing works
Concepts and Strategies in Retinal Gene Therapy
Aguirre, G. D. (2017) · Investigative Ophthalmology & Visual Science · doi:10.1167/iovs.17-22978 · Cited by 24 · see citing works
Sun, S., & Schaffer, D. V. (2018) · Current Opinion in Neurobiology · doi:10.1016/j.conb.2017.12.011 · Cited by 23 · see citing works
Sarkis, S., Chamard, C., Johansen, B., Daïen, V., & Michon, F. (2025) · Frontiers in Medicine · doi:10.3389/fmed.2025.1527319 · Cited by 24 · see citing works
Kovacs, K. D., Ciulla, T. A., & Kiss, S. (2022) · Expert Opinion on Biological Therapy · doi:10.1080/14712598.2022.2121646 · Cited by 20 · see citing works
Early-Onset Progressive Degeneration of the Area Centralis in RPE65-Deficient Dogs
Mowat, F. M., Gervais, K. J., Occelli, L. M., Annear, M. J., Querubin, J. R., Bainbridge, J. W., Smith, A. J., Ali, R. R., & Petersen‐Jones, S. M. (2017) · Investigative Ophthalmology & Visual Science · doi:10.1167/iovs.17-21930 · Cited by 20 · see citing works
Self-citationGene Therapy to the Retina and the Cochlea
Crane, R., Conley, S. M., Al‐Ubaidi, M. R., & Naash, M. I. (2021) · Frontiers in Neuroscience · doi:10.3389/fnins.2021.652215 · Cited by 23 · see citing works
Antiangiogenic Eye Gene Therapy
Corydon, T. J. (2015) · Human Gene Therapy · doi:10.1089/hum.2015.064 · Cited by 18 · see citing works
Lee, S. H., Kim, Y.-S., Nah, S. K., Kim, H. J., Park, H. Y., Yang, J. Y., Park, K., & Park, T. K. (2018) · Molecular Therapy — Methods & Clinical Development · doi:10.1016/j.omtm.2018.01.008 · Cited by 18 · see citing works
Retina transduction by rAAV2 after intravitreal injection: Comparison between mouse and rat
Dias, M. S., Araujo, V. G., Vasconcelos, T., Li, Q., Hauswirth, W. W., Linden, R., & Petrs-Silva, H. (2019) · Gene Therapy · doi:10.1038/s41434-019-0100-9 · Cited by 17 · see citing works
Self-citationSong, H., Bush, R. A., Zeng, Y., Qian, H., Wu, Z., & Sieving, P. A. (2019) · Molecular Therapy - Methods & Clinical Development · doi:10.1016/j.omtm.2018.12.006 · Cited by 17 · see citing works
Annamalai, B., Parsons, N., Nicholson, C., Obert, E., Jones, B., & Rohrer, B. (2021) · Investigative Opthalmology & Visual Science · doi:10.1167/iovs.62.4.11 · Cited by 16 · see citing works
Ross, M., Obolensky, A., Averbukh, E., Ezra-Elia, R., Yamin, E., Honig, H., Dvir, H., Rosov, A., Hauswirth, W. W., Gootwine, E., et al. (2020) · Human Gene Therapy · doi:10.1089/hum.2020.023 · Cited by 16 · see citing works
Self-citationRetinal gene therapy: current progress and future prospects
Ku, C. A., & Pennesi, M. E. (2015) · Expert Review of Ophthalmology · doi:10.1586/17469899.2015.1035711 · Cited by 16 · see citing works
Advancing precision medicines for ocular disorders: Diagnostic genomics to tailored therapies
Panikker, P., Roy, S., Ghosh, A., Poornachandra, B., & Ghosh, A. (2022) · Frontiers in Medicine · doi:10.3389/fmed.2022.906482 · Cited by 16 · see citing works
Development of a translatable gene augmentation therapy for CNGB1-retinitis pigmentosa
Occelli, L. M., Zobel, L., Stoddard, J., Wagner, J., Pasmanter, N., Querubin, J. R., Renner, L. M., Reynaga, R., Winkler, P. A., Sun, K., et al. (2023) · Molecular Therapy · doi:10.1016/j.ymthe.2023.04.005 · Cited by 14 · see citing works
Self-citationRoss, M., Obolensky, A., Averbukh, E., Desrosiers, M., Ezra-Elia, R., Honig, H., Yamin, E., Rosov, A., Dvir, H., Gootwine, E., et al. (2022) · Gene Therapy · doi:10.1038/s41434-021-00306-1 · Cited by 13 · see citing works
AAVrh-10 transduces outer retinal cells in rodents and rabbits following intravitreal administration
Zeng, Y., Qian, H., Wu, Z., Marangoni, D., Sieving, P. A., & Bush, R. A. (2019) · Gene Therapy · doi:10.1038/s41434-019-0094-3 · Cited by 12 · see citing works
Tian, X., Zheng, Q., Xie, J., Zhou, Q., Liang, L., Xu, G., Chen, H., Ling, C., & Lu, D. (2023) · Molecular Therapy — Nucleic Acids · doi:10.1016/j.omtn.2023.05.001 · Cited by 12 · see citing works
Challenges in AAV-Based Retinal Gene Therapies and the Role of Magnetic Nanoparticle Platforms
Siontas, O., & Ahn, S. (2024) · Journal of Clinical Medicine · doi:10.3390/jcm13237385 · Cited by 12 · see citing works
Kick, G. R., Whiting, R. E. H., Ota‐Kuroki, J., Castaner, L. J., Morgan-Jack, B. R., Sabol, J. C., Meiman, E. J., Ortiz, F., & Katz, M. L. (2022) · Experimental Eye Research · doi:10.1016/j.exer.2022.109344 · Cited by 12 · see citing works
Hareendran, S., Ramakrishna, B., & Jayandharan, G. R. (2016) · European Journal of Immunology · doi:10.1002/eji.201545867 · Cited by 10 · see citing works
Peripapillary Intravitreal Injection Improves AAV-Mediated Retinal Transduction
Madrakhimov, S. B., Yang, J. Y., Ahn, D. H., Han, J. W., Ha, T. H., & Park, T. K. (2020) · Molecular Therapy — Methods & Clinical Development · doi:10.1016/j.omtm.2020.03.018 · Cited by 10 · see citing works
Minella, A., Mowat, F. M., Willett, K. L., Sledge, D. G., Bartoe, J. T., Bennett, J., & Petersen‐Jones, S. M. (2014) · Gene Therapy · doi:10.1038/gt.2014.65 · Cited by 9 · see citing works
Self-citationTan, J., Zhang, X., Li, D., Liu, G., Wang, Y., Zhang, D., Wang, X., Tian, W., Dong, X., Zhou, L., et al. (2020) · Molecular Therapy — Methods & Clinical Development · doi:10.1016/j.omtm.2020.04.014 · Cited by 9 · see citing works
Occelli, L. M., Pirie, C. G., & Petersen‐Jones, S. M. (2022) · Veterinary Ophthalmology · doi:10.1111/vop.12973 · Cited by 8 · see citing works
Self-citationOh, A., Loew, E. R., Foster, M. L., Davidson, M. G., English, R. V., Gervais, K. J., Herring, I. P., & Mowat, F. M. (2018) · Documenta Ophthalmologica · doi:10.1007/s10633-018-9653-y · Cited by 8 · see citing works
Self-citation“Para-retinal” Vector Administration into the Deep Vitreous Enhances Retinal Transgene Expression
Zeng, Y., Boyd, R. F., Bartoe, J. T., Wiley, H. E., Marangoni, D., Wei, L. L., & Sieving, P. A. (2020) · Molecular Therapy — Methods & Clinical Development · doi:10.1016/j.omtm.2020.06.015 · Cited by 7 · see citing works
Self-citationTang, M., Zhong, L., Rong, H., Li, K., Ye, M., Peng, J., & Ge, J. (2024) · Experimental Eye Research · doi:10.1016/j.exer.2024.109931 · Cited by 6 · see citing works
Zhang, H., Sajdak, B. S., Merriman, D. K., Carroll, J., & Lipinski, D. M. (2021) · Molecular Therapy — Methods & Clinical Development · doi:10.1016/j.omtm.2021.06.005 · Cited by 6 · see citing works
Viral Vector Technologies and Strategies: Improving on Nature
Croze, R. H., Kotterman, M. A., Burns, C. H., Schmitt, C. E., Quezada, M., Schaffer, D. V., Kirn, D. H., & Francis, P. J. (2021) · International Ophthalmology Clinics · doi:10.1097/iio.0000000000000361 · Cited by 5 · see citing works
Gene Therapy for Inherited Retinopathies: Update on Development Progress
Sun, S. Y., & Montezuma, S. R. (2018) · Journal of VitreoRetinal Diseases · doi:10.1177/2474126418783934 · Cited by 5 · see citing works
Murray, S. J., & Mitchell, N. L. (2022) · Scientific Reports · doi:10.1038/s41598-022-07612-7 · Cited by 5 · see citing works
Delivery of Genetic Information: Viral Vector and Nonviral Vector Gene Therapies
Gemayel, M. C., Byrne, L. C., Ciulla, T. A., & Sahel, J. (2021) · International Ophthalmology Clinics · doi:10.1097/iio.0000000000000360 · Cited by 4 · see citing works
DNA- and RNA-based Gene Therapies in Ophthalmology
Ciulla, T. A., Pennesi, M. E., Kiss, S., & Cunningham, E. T. (2021) · International Ophthalmology Clinics · doi:10.1097/iio.0000000000000359 · Cited by 4 · see citing works
Tripathi, S., Huda, S., Kar, J., Chandra, D., Jayandharan, G. R., & Mohan, N. (2025) · Journal of Cell Science · doi:10.1242/jcs.264190 · Cited by 3 · see citing works
Intravitreal AAV2 gene delivery to feline retinal ganglion cells
Oikawa, K., Eaton, J. S., Kiland, J. A., Torné, O., Mathu, V., Nickells, R. W., & McLellan, G. J. (2024) · Vision Research · doi:10.1016/j.visres.2024.108519 · Cited by 3 · see citing works
Genetic Variants and Impact in PDE6B Rod-Cone Dystrophy
Watson, C. J. G., Nash, B. M., Loi, T. H., Grigg, J., & Jamieson, R. V. (2021) · Essentials in ophthalmology/Essentials ophthalmology · doi:10.1007/978-981-15-9184-6_15 · Cited by 2 · see citing works
Metabolic changes and retinal remodeling in Heterozygous CRX mutant cats (CRX)
Occelli, L. M., Jones, B. W., Cervantes, T. J., & Petersen‐Jones, S. M. (2023) · Experimental Eye Research · doi:10.1016/j.exer.2023.109630 · Cited by 2 · see citing works
Self-citationVirus Vectors for Optogenetic Prosthetization of the Retina
Rotov, A. Y., Nikolaeva, D. A., Astakhova, L. A., & Firsov, M. L. (2020) · Neuroscience and Behavioral Physiology · doi:10.1007/s11055-020-00911-4 · Cited by 2 · see citing works
Chromophore-loaded CRALBP mutant proteins restore rod function in chromophore-deficient mice
Kolesnikov, A. V., Aeschimann, W., Cullity, J., Halabi, M., Kiser, P. D., Stocker, A., & Kefalov, V. J. (2026) · Molecular Therapy Advances · doi:10.1016/j.omta.2026.201701 · Cited by 1 · see citing works
Cui, M., Liu, H., Cai, L., Zhang, Q., Yuan, L., Gao, C., Li, C., Xi, J., Li, Y., Wu, C., et al. (2026) · Molecular Therapy Advances · doi:10.1016/j.omta.2026.201806 · Cited by 1 · see citing works
Michalakis, S., Gerhardt, M.-J., Priglinger, C., & Priglinger, S. G. (2021) · Springer eBooks · doi:10.1007/978-3-319-90495-5_150-1 · Cited by 1 · see citing works
Glaucoma - Recent Advances and New Perspectives
Davey, P. G. (2022) · IntechOpen eBooks · doi:10.5772/intechopen.98102 · Cited by 1 · see citing works
Luo, L., Xu, J., Wang, B.-Q., Chen, C., Chen, X., Hu, Q., Wang, Y., Zhang, W.-Y., Jiang, W., Li, X.-T., et al. (2023) · Biomaterials · doi:10.1016/j.biomaterials.2023.122403 · Cited by 1 · see citing works
Michalakis, S., Gerhardt, M.-J., Priglinger, C., & Priglinger, S. G. (2022) · Springer eBooks · doi:10.1007/978-3-030-42634-7_150 · Cited by 1 · see citing works
Perspective on Gene Therapy for Glaucoma
Dias, M. S., Araújo, V. G., Lani‐Louzada, R., Linden, R., Ribas, V. T., & Petrs‐Silva, H. (2022) · IntechOpen eBooks · doi:10.5772/intechopen.104295 · Cited by 1 · see citing works
- 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
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › 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
About these checks
Registry, retraction, Cites and statistics results are computed, not judged, with one exception: a printed reference that no index lists is read once by a model before it counts as a citation error. Claims are extracted by a model and kept only when their quote appears verbatim in the text; each cited source is then checked, with a model’s help, for whether it says what the claim relies on. Those verdicts can be wrong — read the claim’s quote and the cited source before relying on one.
Statistics and arithmetic are recomputed, not judged.
Confidence bands are shown only for fields calibrated on our annotated test set. None is calibrated yet, so no band is shown.
What counts as a claim ›How we measure confidence ›How we check this ›
Are you an author, or do you see a problem with this page? Request a review
