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Review of gene therapies for age-related macular degeneration
Open accessAbstract
Gene therapies aim to deliver a therapeutic payload to specified tissues with underlying protein deficiency. Since the 1990s, gene therapies have been explored as potential treatments for chronic conditions requiring lifetime care and medical management. Ocular gene therapies target a range of ocular disorders, but retinal diseases are of particular importance due to the prevalence of retinal disease and the current treatment burden of such diseases on affected patients, as well as the challenge of properly delivering these therapies to the target tissue. The purpose of this review is to provide an update on the most current data available for five different retinal gene therapies currently undergoing clinical trials for use against age-related macular degeneration (AMD) and the development of novel delivery routes for the administration of such therapies. Research has been performed and compiled from PubMed and the select authors of this manuscript on the treatment and effectiveness of five current retinal gene therapies: Luxturna, ADVM-022, RGX-314, GT-005, and HMR59. We present the available data of current clinical trials for the treatment of neovascular and dry age-related macular degeneration with different AAV-based gene therapies. We also present current research on the progress of developing novel routes of administration for ocular gene therapies. Retinal gene therapies offer the potential for life-changing treatment for chronic conditions like age-related macular degeneration with a single administration. In doing so, gene therapies change the landscape of treatment options for these chronic conditions for both patient and provider.
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“Gene therapy, successfully used in rare, monogenetic disorders, may prove to be a durable management approach for common, polygenetic conditions, including neovascular age-related macular degeneration (nAMD).”
From Safety and efficacy of ixoberogene soroparvovec in neovascular age-related macular degeneration in the United States (OPTIC): a prospective, two-year, multicentre phase 1 study (2024) · cites this paper as [3]
Experimental — accuracy not yet measuredThe source backs part of the claim, not all of it — or backs it for a different population, condition or comparison.
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“Gene therapy for retinal disease is especially promising since most inherited retinal disorders (IRDs) are monogenetic, cells are post-mitotic, and the imaging modalities used for diagnosis and monitoring are well-described and non-invasive.2,3”
From Safety and efficacy of ixoberogene soroparvovec in neovascular age-related macular degeneration in the United States (OPTIC): a prospective, two-year, multicentre phase 1 study (2024) · cites this paper as [3]
Experimental — accuracy not yet measuredThe source backs part of the claim, not all of it — or backs it for a different population, condition or comparison.
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“Due to its relative immune privilege, the eye represents an ideal environment to deliver viral vector-based in vivo gene therapy via intravitreal, subretinal, or suprachoroidal delivery.2,3”
From Safety and efficacy of ixoberogene soroparvovec in neovascular age-related macular degeneration in the United States (OPTIC): a prospective, two-year, multicentre phase 1 study (2024) · cites this paper as [3]
Experimental — accuracy not yet measuredThe source backs part of the claim, not all of it — or backs it for a different population, condition or comparison.
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“Aflibercept is an anti-VEGF recombinant fusion protein, which was FDA approved for nAMD in 2011 and has been administered intravitreally over 20 million times worldwide to treat VEGF-mediated retinal disorders.3,7”
From Safety and efficacy of ixoberogene soroparvovec in neovascular age-related macular degeneration in the United States (OPTIC): a prospective, two-year, multicentre phase 1 study (2024) · cites this paper as [3]
Experimental — accuracy not yet measuredIn the citing paper
“Several ocular gene therapies are in development for disorders affecting individuals older than 65 years (e.g., age-related macular degeneration [AMD]).27,28”
From Characterization of the ocular inflammatory response to AAV reveals divergence by sex and age (2025) · cites this paper as [28]
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Citations
Cites
Programmable editing of a target base in genomic DNA without double-stranded DNA cleavage
Komor, A. C., Kim, Y. B., Packer, M. S., Zuris, J. A., & Liu, D. R. (2016) · Nature · doi:10.1038/nature17946 · Cited by 5,437
Search-and-replace genome editing without double-strand breaks or donor DNA
Anzalone, A. V., Randolph, P. B., Davis, J. R., Sousa, A. A., Koblan, L. W., Levy, J. M., Chen, P. J., Wilson, C., Newby, G. A., Raguram, A., et al. (2019) · Nature · doi:10.1038/s41586-019-1711-4 · Cited by 4,767
Programmable base editing of A•T to G•C in genomic DNA without DNA cleavage
Gaudelli, N. M., Komor, A. C., Rees, H. A., Packer, M. S., Badran, A. H., Bryson, D. I., & Liu, D. R. (2017) · Nature · doi:10.1038/nature24644 · Cited by 4,184
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
Russell, 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
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
A serious adverse event after successful gene therapy for X-linked severe combined immunodeficiency
Hacein-Bey-Abina, S., von Kalle, C., Schmidt, M., Le Deist, F., Wulffraat, N., McIntyre, E., Radford, I., Villeval, J.-L., Fraser, C. C., Cavazzana-Calvo, M., et al. (2003) · New England Journal of Medicine · doi:10.1056/nejm200301163480314 · Cited by 1,867 · cited 1×
“In 1999, gene therapy for the treatment of severe combined immunodeficiency-X1 ultimately resulted in the death of one patient from unforeseen oncogene activation [4].”
BackgroundT Lymphocyte-Directed Gene Therapy for ADA − SCID: Initial Trial Results After 4 Years
Blaese, R. M., Culver, K. W., Miller, A. D., Carter, C. S., Fleisher, T. A., Clerici, M., Shearer, G. M., Chang, L., Chiang, Y. L., Tolstoshev, P., et al. (1995) · Science · doi:10.1126/science.270.5235.475 · Cited by 1,546
Dunbar, C. E., High, K. A., Joung, J. K., Kohn, D. B., Ozawa, K., & Sadelain, M. (2018) · Science · doi:10.1126/science.aan4672 · Cited by 1,410
Adeno-Associated Virus (AAV) as a Vector for Gene Therapy
Naso, M. F., Tomkowicz, B., Perry, W. L., & Strohl, W. R. (2017) · BioDrugs · doi:10.1007/s40259-017-0234-5 · Cited by 1,362
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
Xiao, X., Li, J., & Samulski, R. J. (1996) · Journal of Virology · doi:10.1128/jvi.70.11.8098-8108.1996 · Cited by 913
Site-specific integration by adeno-associated virus.
Kotin, R. M., Siniscalco, M., Samulski, R. J., Zhu, X. D., Hunter, L., Laughlin, C. A., McLaughlin, S., Muzyczka, N., Rocchi, M., & Berns, K. I. (1990) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.87.6.2211 · Cited by 906
Gene Therapy for Human Genetic Disease?
Friedmann, T., & Roblin, R. O. (1972) · Science · doi:10.1126/science.175.4025.949 · Cited by 819
Long-Term Effect of Gene Therapy on Leber’s Congenital Amaurosis
Bainbridge, J. W. B., Mehat, M. S., Sundaram, V., Robbie, S. J., Barker, S. E., Ripamonti, C., Georgiadis, A., Mowat, F. M., Beattie, S. G., Gardner, P. J., et al. (2015) · New England Journal of Medicine · doi:10.1056/nejmoa1414221 · Cited by 743
Mutations in the CEP290 (NPHP6) Gene Are a Frequent Cause of Leber Congenital Amaurosis
den Hollander, A. I., Koenekoop, R. K., Yzer, S., López, I., Arends, M. L., Voesenek, K., Zonneveld, M. N., Strom, T. M., Meitinger, T., Brunner, H. G., et al. (2006) · The American Journal of Human Genetics · doi:10.1086/507318 · Cited by 669
Dystrophin Immunity in Duchenne's Muscular Dystrophy
Mendell, J. R., Campbell, K., Rodino-Klapac, L., Sahenk, Z., Shilling, C., Lewis, S., Bowles, D., Gray, S., Li, C., Galloway, G., et al. (2010) · New England Journal of Medicine · doi:10.1056/nejmoa1000228 · Cited by 655
Immune responses to gene therapy vectors: influence on vector function and effector mechanisms
Bessis, N., Garcia-Cozar, F. J., & Boissier, M. (2004) · Gene Therapy · doi:10.1038/sj.gt.3302364 · Cited by 653
Drusen complement components C3a and C5a promote choroidal neovascularization
Nozaki, M., Raisler, B. J., Sakurai, E., Sarma, J. V., Barnum, S. R., Lambris, J. D., Chen, Y., Zhang, K., Ambati, B. K., Baffi, J. Z., et al. (2006) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.0408835103 · Cited by 650
Jacobson, S. G. (2012) · Archives of Ophthalmology · doi:10.1001/archophthalmol.2011.298 · Cited by 639
Kessler, P., Podsakoff, G. M., Chen, X., McQuiston, S. A., Colosi, P., Matelis, L. A., Kurtzman, G. J., & Byrne, B. J. (1996) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.93.24.14082 · Cited by 614
Bennett, J., Wellman, J., Marshall, K. A., McCague, S., Ashtari, M., DiStefano-Pappas, J., Elci, O. U., Chung, D. C., Sun, J., Wright, J. F., et al. (2016) · The Lancet · doi:10.1016/s0140-6736(16)30371-3 · Cited by 461
Maguire, A. M., Russell, S., Wellman, J. A., Chung, D. C., Yu, Z.-F., Tillman, A., Wittes, J., Pappas, J., Elci, O., Marshall, K. A., et al. (2019) · Ophthalmology · doi:10.1016/j.ophtha.2019.06.017 · Cited by 409
Differential activation of innate immune responses by adenovirus and adeno-associated virus vectors
Zaiss, A.-K., Liu, Q., Bowen, G. P., Wong, N. C. W., Bartlett, J. S., & Muruve, D. A. (2002) · Journal of Virology · doi:10.1128/jvi.76.9.4580-4590.2002 · Cited by 394
Keeler, A. M., & Flotte, T. R. (2019) · Annual Review of Virology · doi:10.1146/annurev-virology-092818-015530 · Cited by 322
Genotoxicity of Retroviral Integration In Hematopoietic Cells
Nienhuis, A. W., Dunbar, C. E., & Sorrentino, B. P. (2006) · Molecular Therapy · doi:10.1016/j.ymthe.2006.03.001 · Cited by 301
CRISPR/Cas9-Mediated Genome Editing as a Therapeutic Approach for Leber Congenital Amaurosis 10
Ruan, G.-X., Barry, E., Yu, D., Lukason, M., Cheng, S. H., & Scaria, A. (2017) · Molecular Therapy · doi:10.1016/j.ymthe.2016.12.006 · Cited by 270
AAV cis -regulatory sequences are correlated with ocular toxicity
Xiong, W., Wu, D. M., Xue, Y., Wang, S. K., Chung, M. J., Ji, X., Rana, P., Zhao, S. R., Mai, S., & Cepko, C. L. (2019) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.1821000116 · Cited by 249
Unraveling the complex story of immune responses to AAV vectors trial after trial.
Vandamme, C., Adjali, O., & Mingozzi, F. (2017) · Human Gene Therapy · doi:10.1089/hum.2017.150 · Cited by 234
Heier, J. S., Kherani, S., Desai, S., Dugel, P., Kaushal, S., Cheng, S. H., Delacono, C., Purvis, A., Richards, S., Le-Halpere, A., et al. (2017) · The Lancet · doi:10.1016/s0140-6736(17)30979-0 · Cited by 229 · cited 1×
“Patients that received the highest doses of intravitreal AAV2-sFlt01 for neovascular AMD (2 × 10(8)–2 × 10(11) vg), or intravitreal AAV2-RS1 for X-linked retinoschisis (1 × 10(10)–1 × 10(11) vg) developed antiAAV2 antibodies and intraocular inflammation that responded to topical or oral steroids [63].”
BackgroundWeleber, R. G., Pennesi, M. E., Wilson, D. J., Kaushal, S., Erker, L. R., Jensen, L., McBride, M. T., Flotte, T. R., Humphries, M., Calcedo, R., et al. (2016) · Ophthalmology · doi:10.1016/j.ophtha.2016.03.003 · Cited by 218
Pharmaceutical development of AAV-based gene therapy products for the eye
Rodrigues, G. A., Shalaev, E., Karami, T. K., Cunningham, J., Slater, N. K. H., & Rivers, H. M. (2019) · Pharmaceutical Research · doi:10.1007/s11095-018-2554-7 · Cited by 213
Pillay, S., Zou, W., Cheng, F., Puschnik, A. S., Meyer, N., Ganaie, S. S., Deng, X., Wosen, J. E., Davulcu, O., Yan, Z., et al. (2017) · Journal of Virology · doi:10.1128/jvi.00391-17 · Cited by 190
Beneficial effects on vision in patients undergoing retinal gene therapy for choroideremia
Xue, K., Jolly, J. K., Barnard, A. R., Rudenko, A., Salvetti, A. P., Patrício, M. I., Edwards, T. L., Groppe, M., Orlans, H. O., Tolmachova, T., et al. (2018) · Nature Medicine · doi:10.1038/s41591-018-0185-5 · Cited by 185
Subretinal injection: a review on the novel route of therapeutic delivery for vitreoretinal diseases
Peng, Y., Tang, L., & Zhou, Y. (2017) · Ophthalmic Research · doi:10.1159/000479157 · Cited by 175
Novel mobility test to assess functional vision in patients with inherited retinal dystrophies
Chung, D. C., McCague, S., Yu, Z., Thill, S., DiStefano‐Pappas, J., Bennett, J., Cross, D., Marshall, K. A., Wellman, J. A., & High, K. A. (2017) · Clinical and Experimental Ophthalmology · doi:10.1111/ceo.13022 · Cited by 172
Latella, M. C., Salvo, M. T. D., Cocchiarella, F., Benati, D., Grisendi, G., Comitato, A., Marigo, V., & Recchia, A. (2016) · Molecular Therapy — Nucleic Acids · doi:10.1038/mtna.2016.92 · Cited by 168
Yiu, G., Chung, S. H., Mollhoff, I. N., Nguyen, U. T., Thomasy, S. M., Yoo, J., Taraborelli, D., & Noronha, G. (2020) · Molecular Therapy - Methods & Clinical Development · doi:10.1016/j.omtm.2020.01.002 · Cited by 153
Self-citationAAV8-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
Nonintegrating Gene Therapy Vectors
Athanasopoulos, T., Munye, M. M., & Yáñez‐Muñoz, R. J. (2017) · Hematology/Oncology Clinics of North America · doi:10.1016/j.hoc.2017.06.007 · Cited by 134
Gene Therapy: Design and Prospects for Craniofacial Regeneration
Scheller, E. L., & Krebsbach, P. (2009) · Journal of Dental Research · doi:10.1177/0022034509337480 · Cited by 132
Review and update on the molecular basis of Leber congenital amaurosis
Chacon-Camacho, O. F. (2015) · World Journal of Clinical Cases · doi:10.12998/wjcc.v3.i2.112 · Cited by 128
Grishanin, R., Vuillemenot, B., Sharma, P., Keravala, A., Greengard, J., Gelfman, C., Blumenkrantz, M., Lawrence, M., Hu, W., Kiss, S., et al. (2019) · Molecular Therapy · doi:10.1016/j.ymthe.2018.11.003 · Cited by 127
Self-citationTsai, Y.-T., Wu, W.-H., Lee, T.-T., Wu, W.-P., Xu, C. L., Park, K. S., Cui, X., Justus, S., Lin, C.-S., Jauregui, R., et al. (2018) · Ophthalmology · doi:10.1016/j.ophtha.2018.04.001 · Cited by 119
Yiu, G. C., Pecen, P. E., Sarin, N., Chiu, S. J., Farsiu, S., Mruthyunjaya, P., & Toth, C. A. (2013) · JAMA Ophthalmology · doi:10.1001/jamaophthalmol.2013.7288 · Cited by 113
Self-citationSeitz, I. P., Michalakis, S., Wilhelm, B., Reichel, F. F., Ochakovski, G. A., Zrenner, E., Ueffing, M., Biel, M., Wissinger, B., Bartz-Schmidt, K. U., et al. (2017) · Investigative Opthalmology & Visual Science · doi:10.1167/iovs.17-22473 · Cited by 108
Dosage thresholds and influence of transgene cassette in adeno-associated virus–related toxicity
Khabou, H., Cordeau, C., Pacot, L., Fisson, S., & Dalkara, D. (2018) · Human Gene Therapy · doi:10.1089/hum.2018.144 · Cited by 93
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Gao, J., Hussain, R. M., & Weng, C. Y. (2020) · Clinical ophthalmology · doi:10.2147/opth.s231804 · Cited by 80
Self-citationThe suprachoroidal space: from potential space to a space with potential
Moisseiev, E., Loewenstein, A., & Yiu, G. (2016) · Clinical Ophthalmology · doi:10.2147/opth.s89784 · Cited by 76
Self-citationProtective responses to sublytic complement in the retinal pigment epithelium
Tan, L. X., Toops, K. A., & Lakkaraju, A. (2016) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.1523061113 · Cited by 66
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Shen, J., Kim, J., Tzeng, S. Y., Ding, K., Hafiz, Z., Long, D., Wang, J., Green, J. J., & Campochiaro, P. A. (2020) · Science Advances · doi:10.1126/sciadv.aba1606 · Cited by 63
Dreismann, A. K., McClements, M. E., Barnard, A. R., Orhan, E., Hughes, J. P., Lachmann, P. J., & MacLaren, R. E. (2021) · Gene Therapy · doi:10.1038/s41434-021-00239-9 · Cited by 58
Host immune responses after suprachoroidal delivery of AAV8 in nonhuman primate eyes
Chung, S. H., Mollhoff, I. N., Mishra, A., Sin, T.-N., Ngo, T., Ciulla, T., Sieving, P., Thomasy, S. M., & Yiu, G. (2021) · Human Gene Therapy · doi:10.1089/hum.2020.281 · Cited by 57
Self-citationYiu, G. C., Tieu, E., Nguyen, A. T., Wong, B. M., & Smit‐McBride, Z. (2016) · Investigative Ophthalmology & Visual Science · doi:10.1167/iovs.16-20296 · Cited by 55
Self-citationGelfman, C. M., Grishanin, R., Bender, K. O., Nguyen, A., Greengard, J., Sharma, P., Nieves, J., Kiss, S., & Gasmi, M. (2021) · Journal of Ocular Pharmacology and Therapeutics · doi:10.1089/jop.2021.0001 · Cited by 55
Self-citationWilloughby, A. S., Vuong, V. S., Cunefare, D., Farsiu, S., Noronha, G., Danis, R. P., & Yiu, G. C. (2017) · American Journal of Ophthalmology · doi:10.1016/j.ajo.2017.11.020 · Cited by 53
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Self-citationResidual Electroretinograms in Young Leber Congenital Amaurosis Patients with Mutations of AIPL1
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Self-citationKiss, S., Grishanin, R., Nguyen, A., Rosario, R., Greengard, J. S., Nieves, J., Gelfman, C. M., & Gasmi, M. (2020) · Molecular Therapy - Methods & Clinical Development · doi:10.1016/j.omtm.2020.06.007 · Cited by 29
Self-citationde Smet, M. D., Lynch, J. L., Dejneka, N. S., Keane, M., & Khan, I. J. (2018) · Investigative Opthalmology & Visual Science · doi:10.1167/iovs.17-22233 · Cited by 23
Drusen complement components C3a and C5a promote choroidal neovascularization
Nozaki, M., Raisler, B. J., Sakurai, E., Sarma, J. V., Barnum, S. R., Lambris, J. D., Chen, Y., Zhang, K., Ambati, B. K., Baffi, J., et al. (2006) · American Journal of Ophthalmology · doi:10.1016/j.ajo.2006.05.010 · Cited by 22
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Yiu, G. C. (2017) · Ophthalmology Retina · doi:10.1016/j.oret.2017.09.015 · Cited by 12
Self-citationHost immune responses after suprachoroidal delivery of AAV8 in nonhuman primate eyesPreprint
Chung, S. H., Mollhoff, I. N., Mishra, A., et al. (2020) · bioRxiv (Cold Spring Harbor Laboratory) · doi:10.1101/2020.09.25.313676 · Cited by 12
Voretigene Neparvovec in Retinal Diseases: A Review of the Current Clinical Evidence
Gao, J., Hussain, R. M., & Weng, C. Y. (2020) · DOAJ (DOAJ: Directory of Open Access Journals) · Cited by 9
Adeno-associated Virus and AAV Vectors for Gene Delivery
Carter, B., Burstein, H., & PELUSO, R. W. (2003) · CRC Press eBooks · doi:10.1201/9780824758608.ch5 · Cited by 2
Research works printed but not linked: 2
- Khanani AM, Kiss S, Turpcu A, Hoang C, Osborne A; Phase 1 Study of Intravitreal Gene Therapy ADVM-022 for neovascular AMD (OPTIC Trial). Invest. Ophthalmol. Vis. Sci. 2020;61:1154.
- Busbee BG, Boyer DS, Khanani AM, et al; Phase 1 Study of Intravitreal Gene Therapy with ADVM-022 for neovascular AMD (OPTIC Trial). Invest. Ophthalmol. Vis. Sci. 2021;62:352.
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Metadata
- Authors
- Khanani, A. M., Thomas, M. J., Aziz, A. A., Weng, C. Y., Danzig, C. J., Yiu, G., Kiss, S., Waheed, N. K., & Kaiser, P. K.
- Affiliations
- University of Nevada, Reno (US)
- Baylor College of Medicine (US)
- Florida Atlantic University (US)
- University of California, Davis (US)
- NewYork–Presbyterian Hospital (US)
- Tufts University (US)
- Cleveland Clinic (US)
- Corresponding author
- Khanani, Arshad M.
- ORCID
- Venue
- Eye
- Published
- 2022-01-11
- Volume
- 36
- Issue
- 2
- Pages
- 303-311
- ISSN
- 0078-5334 (ISSN-L)
- 0950-222X
- 1476-5454
- PMC
- PMC8807824
- OpenAlex
- W4206710973
- Cited by
- 119
- References
- 71
- Citations by year
- 2026: 15 · 2025: 29 · 2024: 36 · 2023: 32 · 2022: 7
- FWCI
- 2.71
- Type
- journal article
- Language
- en
- Open access
- bronze
- doi.org
- Licence
- Locations
- Eye (publishedVersion)
- PubMed Central (publishedVersion)
- PubMed (publishedVersion)
- Digital Commons-TMC (Texas Medical Center) (submittedVersion)
- eScholarship (California Digital Library) (submittedVersion)
- Topics
- Health Sciences › Medicine › Ophthalmology › Retinal Development and Disorders
- Health Sciences › Medicine › Ophthalmology › Retinal Diseases and Treatments
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Genetics › Virus-based gene therapy research
- Keywords
- age-related macular degeneration, retinal gene therapy, adeno-associated virus vectors, neovascular age-related macular degeneration, dry age-related macular degeneration, Luxturna, clinical trials
- MeSH terms
- Humans, Macular Degeneration, Genetic Therapy, Geographic Atrophy
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 ›
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