Gene therapy for inherited retinal disease: Long-term durability of effect

Safety and Efficacy of Gene Transfer for Leber's Congenital Amaurosis

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Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65 -mediated inherited retinal dystrophy: a randomised, controlled, open-label, phase 3 trial

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Gene therapy restores vision in a canine model of childhood blindness

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Efficient long-term gene transfer into muscle tissue of immunocompetent mice by adeno-associated virus vector

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Emerging issues in AAV-mediated in vivo gene therapy

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Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trial

Maguire, A. M., High, K. A., Auricchio, A., Wright, J. F., Pierce, E. A., Testa, F., Mingozzi, F., Bennicelli, J. L., Ying, G.-S., Rossi, S., et al. (2009) · The Lancet · doi:10.1016/s0140-6736(09)61836-5 · Cited by 865 · see citing works

Long-Term Effect of Gene Therapy on Leber’s Congenital Amaurosis

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Gene Therapy for Leber Congenital Amaurosis Caused by RPE65 Mutations: Safety and Efficacy in 15 Children and Adults Followed Up to 3 Years

Jacobson, S. G. (2012) · Archives of Ophthalmology · doi:10.1001/archophthalmol.2011.298 · Cited by 639 · see citing works

The Retinal Pigment Epithelium in Health and Disease

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FUNDUS AUTOFLUORESCENCE IMAGING

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RPE65 is the isomerohydrolase in the retinoid visual cycle

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Circular Intermediates of Recombinant Adeno-Associated Virus Have Defined Structural Characteristics Responsible for Long-Term Episomal Persistence in Muscle Tissue

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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

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

Safety and durability of effect of contralateral-eye administration of AAV2 gene therapy in patients with childhood-onset blindness caused by RPE65 mutations: a follow-on phase 1 trial

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 · see citing works

Human retinal gene therapy for Leber congenital amaurosis shows advancing retinal degeneration despite enduring visual improvement

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Molecular genetics and emerging therapies for retinitis pigmentosa: Basic research and clinical perspectives

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Mutation of key residues of RPE65 abolishes its enzymatic role as isomerohydrolase in the visual cycle

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Efficacy, Safety, and Durability of Voretigene Neparvovec-rzyl in RPE65 Mutation–Associated Inherited Retinal Dystrophy

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 · cited 7× · see citing works

AAV2 Gene Therapy Readministration in Three Adults with Congenital Blindness

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Improvement and Decline in Vision with Gene Therapy in Childhood Blindness

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Initial results from a first-in-human gene therapy trial on X-linked retinitis pigmentosa caused by mutations in RPGR

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Adeno-Associated Virus Vector Genomes Persist as Episomal Chromatin in Primate Muscle

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Identifying photoreceptors in blind eyes caused by RPE65 mutations: Prerequisite for human gene therapy success

Jacobson, S. G., Alemán, T. S., Cideciyan, A. V., Sumaroka, A., Schwartz, S., Windsor, E. A. M., Traboulsi, E. I., Héon, E., Pittler, S. J., Milam, A. H., et al. (2005) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.0500646102 · Cited by 273 · see citing works

Reversal of Blindness in Animal Models of Leber Congenital Amaurosis Using Optimized AAV2-mediated Gene Transfer

Bennicelli, J., Wright, J. F., Komaromy, A., Jacobs, J. B., Hauck, B., Zelenaia, O., Mingozzi, F., Hui, D., Chung, D., Rex, T. S., et al. (2008) · Molecular Therapy · doi:10.1038/sj.mt.6300389 · Cited by 267 · see citing works

Gene 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

Gene therapy rescues photoreceptor blindness in dogs and paves the way for treating human X-linked retinitis pigmentosa

Beltran, 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

Functional 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 · see citing works

AAV <i>cis</i> -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 · see citing works

Immune responses to retinal gene therapy using adeno-associated viral vectors – implications for treatment success and safety

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Clinical development of gene therapy: results and lessons from recent successes

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Pharmaceutical development of AAV-based gene therapy products for the eye

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Rescue from Photoreceptor Degeneration in the rd Mouse by Human Immunodeficiency Virus Vector-Mediated Gene Transfer

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Restoration of vision in RPE65-deficient Briard dogs using an AAV serotype 4 vector that specifically targets the retinal pigmented epithelium

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

Gene Therapy Restores Vision-Dependent Behavior as Well as Retinal Structure and Function in a Mouse Model of RPE65 Leber Congenital Amaurosis

Pang, J., Chang, B., Kumar, A., Nusinowitz, S., Noorwez, S. M., Li, J., Rani, A., Foster, T. C., Chiodo, V. A., Doyle, T. J., et al. (2005) · Molecular Therapy · doi:10.1016/j.ymthe.2005.09.001 · Cited by 199 · see citing works

In Utero Gene Therapy Rescues Vision in a Murine Model of Congenital Blindness

Dejneka, N. S., Surace, E. M., Alemán, T. S., Cideciyan, A. V., Lyubarsky, A., Savchenko, A., Redmond, T. M., Tang, W., Wei, Z., Rex, T. S., et al. (2004) · Molecular Therapy · doi:10.1016/j.ymthe.2003.11.013 · Cited by 197 · see citing works

Durability of Voretigene Neparvovec for Biallelic RPE65-Mediated Inherited Retinal Disease

Maguire, A. M., Russell, S., Chung, D. C., Yu, Z.-F., Tillman, A., Drack, A. V., Simonelli, F., Leroy, B. P., Reape, K. Z., High, K. A., et al. (2021) · Ophthalmology · doi:10.1016/j.ophtha.2021.03.031 · Cited by 194 · see citing works

Long-term and age-dependent restoration of visual function in a mouse model of CNGB3-associated achromatopsia following gene therapy

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Novel mobility test to assess functional vision in patients with inherited retinal dystrophies

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Designer Gene Delivery Vectors: Molecular Engineering and Evolution of Adeno-Associated Viral Vectors for Enhanced Gene Transfer

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Perifoveal Chorioretinal Atrophy after Subretinal Voretigene Neparvovec-rzyl for RPE65-Mediated Leber Congenital Amaurosis

Gange, W. S., Sisk, R. A., Besirli, C. G., Lee, T. C., Havunjian, M., Schwartz, H., Borchert, M., Sengillo, J. D., Mendoza‐Santiesteban, C. E., Berrocal, A. M., et al. (2021) · Ophthalmology Retina · doi:10.1016/j.oret.2021.03.016 · Cited by 157 · see citing works

The Status ofRPE65Gene Therapy Trials: Safety and EfficacyPreprint

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Safety in Nonhuman Primates of Ocular AAV2- RPE65 , a Candidate Treatment for Blindness in Leber Congenital Amaurosis

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Gene therapy progress and prospects: the eye

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Safety and efficacy of subretinal readministration of a viral vector in large animals to treat congenital blindness

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AAV-Mediated Gene Therapy for Retinal Degeneration in the rd10 Mouse Containing a Recessive PDEβ Mutation

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Gene Therapy 2017: Progress and Future Directions

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The Natural History of Inherited Retinal Dystrophy Due to Biallelic Mutations in the RPE65 Gene

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Gene replacement therapy in the retinal degeneration slow (rds)mouse: the effect on retinal degeneration following partial transduction of the retina

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Long-term Evaluation of AAV-Mediated sFlt-1 Gene Therapy for Ocular Neovascularization in Mice and Monkeys

Lai, C., Shen, W.-Y., Brankov, M., Lai, Y. K. Y., Barnett, N. L., Lee, S., Yeo, I. Y. S., Mathur, R., Ho, J. E., Pineda, P., et al. (2005) · Molecular Therapy · doi:10.1016/j.ymthe.2005.04.022 · Cited by 124 · see citing works

Noninvasive gene delivery to foveal cones for vision restoration

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Changes in Retinal Sensitivity in Geographic Atrophy Progression as Measured by Microperimetry

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AAV-mediated gene therapy for retinal disorders: from mouse to man

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Clinical applications of retinal gene therapy

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In Vivo Gene Therapy in Young and Adult RPE65-/- Dogs Produces Long-Term Visual Improvement

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Lentiviral Gene Transfer of Rpe65 Rescues Survival and Function of Cones in a Mouse Model of Leber Congenital Amaurosis

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Canine and Human Visual Cortex Intact and Responsive Despite Early Retinal Blindness from RPE65 Mutation

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AAV-Mediated Cone Rescue in a Naturally Occurring Mouse Model of CNGA3-Achromatopsia

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Gene Augmentation Therapy Restores Retinal Function and Visual Behavior in a Sheep Model of CNGA3 Achromatopsia

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Taking Stock of Retinal Gene Therapy: Looking Back and Moving Forward

Bennett, J. (2017) · Molecular Therapy · doi:10.1016/j.ymthe.2017.03.008 · Cited by 110 · see citing works

Aging in Mouse and Human Systems: A Comparative Study

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Gene therapy restores vision in rd1 mice after removal of a confounding mutation in Gpr179

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Recombinant adeno-associated virus type 2-mediated gene delivery into the Rpe65 -/- knockout mouse eye results in limited rescue

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Gene therapy restores vision and delays degeneration in the CNGB1-/- mouse model of retinitis pigmentosa

Koch, S., Sothilingam, V., Garrido, M. G., Tanimoto, N., Bećirović, E., Koch, F., Seide, C., Beck, S. C., Seeliger, M. W., Biel, M., et al. (2012) · Human Molecular Genetics · doi:10.1093/hmg/dds290 · Cited by 96 · see citing works

Dosage thresholds and influence of transgene cassette in adeno-associated virus–related toxicity

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Successful arrest of photoreceptor and vision loss expands the therapeutic window of retinal gene therapy to later stages of disease

Beltran, W. A., Cideciyan, A. V., Iwabe, S., Świder, M., Kosyk, M. S., McDaid, K. S., Martynyuk, I., Ying, G., Shaffer, J., Deng, W., et al. (2015) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.1509914112 · Cited by 90 · see citing works

Halting progressive neurodegeneration in advanced retinitis pigmentosa

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Cone-Specific Promoters for Gene Therapy of Achromatopsia and Other Retinal Diseases

Ye, G., Budzynski, E., Sonnentag, P. J., Nork, T. M., Sheibani, N., Gürel, Z., Boye, S. L., Peterson, J. J., Boye, S. L., Hauswirth, W. W., et al. (2015) · Human Gene Therapy · doi:10.1089/hum.2015.130 · Cited by 85 · see citing works

Transient Photoreceptor Deconstruction by CNTF Enhances rAAV-Mediated Cone Functional Rescue in Late Stage CNGB3-Achromatopsia

Komáromy, A. M., Rowlan, J. S., Corr, A. T. P., Reinstein, S. L., Boye, S. L., Cooper, A. E., Gonzalez, A., Levy, B. J., Wen, R., Hauswirth, W. W., et al. (2013) · Molecular Therapy · doi:10.1038/mt.2013.50 · Cited by 82 · see citing works

Advances in gene therapy for diseases of the eye

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Optimization of Retinal Gene Therapy for X-Linked Retinitis Pigmentosa Due to RPGR Mutations

Beltran, W. A., Cideciyan, A. V., Boye, S. E., Ye, G.-J., Iwabe, S., Dufour, V. L., Marinho, L. F., Swider, M., Kosyk, M. S., Sha, J., et al. (2017) · Molecular Therapy · doi:10.1016/j.ymthe.2017.05.004 · Cited by 82 · see citing works

Photoreceptor rescue by an abbreviated human RPGR gene in a murine model of X-linked retinitis pigmentosa

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Replacement Gene Therapy with a Human RPGRIP1 Sequence Slows Photoreceptor Degeneration in a Murine Model of Leber Congenital Amaurosis

Pawlyk, B. S., Bulgakov, O. V., Liu, X., Xu, X., Adamian, M., Sun, X., Khani, S. C., Berson, E. L., Sandberg, M. A., & Li, T. (2010) · Human Gene Therapy · doi:10.1089/hum.2009.218 · Cited by 78 · see citing works

A long-term efficacy study of gene replacement therapy for RPGR-associated retinal degeneration

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Prospect of retinal gene therapy following commercialization of voretigene neparvovec-rzyl for retinal dystrophy mediated by RPE65 mutation

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Long-term Structural Outcomes of Late-stage RPE65 Gene Therapy

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Optogenetics and Targeted Gene Therapy for Retinal Diseases: Unravelling the Fundamentals, Applications, and Future Perspectives

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Gene therapy strategies for aging intervention

Jing, Y., Ren, J., Qu, J. T., & Liu, G. (2025) · Cell Insight · doi:10.1016/j.cellin.2025.100254 · Cited by 10 · see citing works

Options for Topical Treatment of Oxidative Eye Diseases with a Special Focus on Retinopathies

Russo, C., Rusciano, D., Santangelo, R., & Malaguarnera, L. (2024) · Medicina · doi:10.3390/medicina60030354 · Cited by 9 · see citing works

AAV-NDI1 Therapy Provides Significant Benefit to Murine and Cellular Models of Glaucoma

Millington‐Ward, S., Palfi, A., Shortall, C., Finnegan, L. K., Bargroff, E., Post, I. J. M., Maguire, J., Irnaten, M., O'BRIEN, C., Kenna, P. F., et al. (2024) · International Journal of Molecular Sciences · doi:10.3390/ijms25168876 · Cited by 8 · see citing works

Addressing Challenges in Developing Treatments for Inherited Retinal Diseases: Recommendations From the Third Monaciano Symposium

Thompson, D. A., Jayasundera, T., Alekseev, O., Ali, R. R., Amato, A., Arshavsky, V. Y., Audo, I. S., Auricchio, A., Bainbridge, J. W., Banin, E., et al. (2025) · Translational Vision Science & Technology · doi:10.1167/tvst.14.8.37 · Cited by 7 · see citing works

Application of Nanomaterials in the Diagnosis and Treatment of Retinal Diseases

Yao, Y., Cao, Q., Fang, H., & Tian, H. (2025) · Small · doi:10.1002/smll.202503070 · Cited by 7 · see citing works

Innovative therapies for inherited retinal dystrophies: navigating DNA, RNA, and protein approaches

Gómez-Escribano, A. P., García-García, G., Pérez-Santamarina, E., Aller-Mañas, E., Vázquez-Manrique, R. P., & Millán-Salvador, J. M. (2025) · eBioMedicine · doi:10.1016/j.ebiom.2025.105751 · Cited by 6 · see citing works

Efficacy and Safety of Voretigene Neparvovec in RPE65-Retinopathy: Results of a Phase III Trial in Japan

Fujinami, K., Akiyama, K., Tsunoda, K., Ito, S., Seko, N., & Yamamoto, S. (2025) · Ophthalmology Science · doi:10.1016/j.xops.2025.100876 · Cited by 6 · see citing works

Hearing loss: a global view for gene therapy approaches and challenges

Elbagoury, N. M. (2025) · European Journal of Pediatrics · doi:10.1007/s00431-025-06426-9 · Cited by 5 · see citing works

Safety of Human USH1C Transgene Expression Following Subretinal Injection in Wild-Type Pigs

Király, P., Klein, J., Seitz, I. P., Reichel, F. F., Peters, T. M., Ardan, T., Juhásová, J., Juhás, Š., Ellederová, Z., Nemesh, Y., et al. (2025) · Investigative Ophthalmology & Visual Science · doi:10.1167/iovs.66.1.48 · Cited by 5 · see citing works

Reviving a Classic Antigen with a Cutting-Edge Approach: Nanobodies for HER2+ Breast Cancer

Castrignano, C., Scipio, F. D., Franco, F., Mognetti, B., & Berta, G. N. (2023) · Pharmaceuticals · doi:10.3390/ph16060794 · Cited by 5 · see citing works

Selective Block of Upregulated Kv1.3 Potassium Channels in ON-Bipolar Cells of the Blind Retina Enhances Optogenetically Restored Signaling

Schilardi, G., Kralik, J., & Kleinlogel, S. (2023) · International Journal of Molecular Sciences · doi:10.3390/ijms241814207 · Cited by 5 · see citing works

Foveal detachment in voretigene neparvovec administration: essential step or avoidable risk?

Király, P., Ivanova, T., & Jalil, A. (2025) · Eye · doi:10.1038/s41433-025-03672-x · Cited by 4 · see citing works

Long-term experience with gene augmentation therapy in patients with inherited retinal disease associated with biallelic mutations in RPE65

Lorenz, B. (2025) · Medizinische Genetik · doi:10.1515/medgen-2024-2067 · Cited by 4 · see citing works

Ocular Delivery of Viral Vectors for Retinal and Choroidal Vascular Diseases: A Review

Agrawal, R., Oraskovich, S. V., Francis, P. J., & Schaffer, D. V. (2025) · American Journal of Ophthalmology · doi:10.1016/j.ajo.2025.07.025 · Cited by 6 · see citing works

Recovery of cone-mediated vision in Lebercilin associated retinal ciliopathy after gene therapy: One-year results of a phase I/II trial

Alemán, T. S., Uyhazi, K. E., Román, A. J., Weber, M. L., O'Neil, E. C., Świder, M., Sumaroka, A., Maguire, K. H., Aleman, E. M., Santos, A. J., et al. (2025) · Molecular Therapy · doi:10.1016/j.ymthe.2025.06.035 · Cited by 4 · see citing works

Intravitreal Delivery of rAAV2-hSyn-hRS1 Results in Retinal Ganglion Cell-Specific Gene Expression and Retinal Improvement in the Rs1 -KO Mouse

Zheng, Y., Xu, X., Fan, R., Jiang, H., Guo, Q., Han, X., Liu, Y., & Luo, G. (2024) · Human Gene Therapy · doi:10.1089/hum.2023.209 · Cited by 4 · see citing works

Genetics and eye health: research advances and implications for primary eyecare

Britten‐Jones, A. C., Knight, L. S. W., Gocuk, S. A., Truong, P., Cichello, E., Amarasekera, S. S. C., Nguyen, B. N., & Ayton, L. N. (2025) · Clinical and Experimental Optometry · doi:10.1080/08164622.2025.2580576 · Cited by 3 · see citing works

Gene therapy for hearing loss: challenges and the promise of cellular plasticity and epigenetic modulation

Das, S., & Manor, U. (2024) · Frontiers in Neurology · doi:10.3389/fneur.2024.1511938 · Cited by 3 · see citing works

The RLR intrinsic antiviral system is expressed in neural retina and restricts lentiviral transduction of human Mueller cells

Sauter, M. M., Noel, H., & Brandt, C. R. (2023) · Experimental Eye Research · doi:10.1016/j.exer.2023.109647 · Cited by 3 · see citing works

AAV2-mediated intravitreal delivery of exon-specific U1 snRNA rescues optic neuropathy in familial dysautonomia

Chekuri, A. K., Kondabolu, K., Kirchner, E. G., Koli, S., Chagnon, M., Krasniqi, D., Stern, M. E., Bolduc, J., Romano, G., Pagani, F., et al. (2025) · Molecular Therapy — Nucleic Acids · doi:10.1016/j.omtn.2025.102788 · Cited by 3 · see citing works

Single-Eye Gene Therapy for Leber Hereditary Optic Neuropathy

Scholl, H. P. N., & György, B. (2024) · JAMA Ophthalmology · doi:10.1001/jamaophthalmol.2024.5618 · Cited by 2 · see citing works

Gene therapy advances using canine and feline animal models of inherited retinal degeneration

Petersen‐Jones, S. M., & Beckwith‐Cohen, B. (2025) · Eye · doi:10.1038/s41433-025-03825-y · Cited by 2 · see citing works

Genetic Therapy of Fuchs Endothelial Corneal Dystrophy: Where Are We? A Review

Pukl, S. S. (2025) · Genes · doi:10.3390/genes16101222 · Cited by 2 · see citing works

Precision gene therapy: Tailoring rAAV-mediated gene therapies for inherited retinal dystrophies (IRDs)

Duarte, F. T., & Arsenijévic, Y. (2025) · Molecular Aspects of Medicine · doi:10.1016/j.mam.2025.101424 · Cited by 2 · see citing works

Keep an Eye on Next Generation Sequencing (NGS) Technology: Secondary Findings and Differential Diagnosis in Inherited Retinal Dystrophies (IRDs)

D’Esposito, F., Capobianco, M., Gagliano, C., Avitabile, A., Gagliano, G., Esposito, G., Dammino, E., Carotenuto, A., & Zeppieri, M. (2025) · Biomedicines · doi:10.3390/biomedicines13051117 · Cited by 2 · see citing works

Advances and challenges of targeted protein degradation in ophthalmology: Future directions and therapeutic potential

Feng, K., Wei, M., Ma, P., Hu, J., Huang, C., Han, Y., & Liu, Z. (2025) · Materials & Design · doi:10.1016/j.matdes.2025.114767 · Cited by 1 · see citing works

Eye‐Targeted A20 Gene Therapy Alleviates Ischemic Retinopathy by Reducing Pathologic Neovascularization, Gliosis, and Neuronal Apoptosis

Essayagh, S., Aridi, T. G., Turunen, T., Choi, L. Y., Adam, H., Patel, N., Scali, S. T., Fisher, M. D., Skaf, K., Csizmadia, E., et al. (2025) · The FASEB Journal · doi:10.1096/fj.202500419r · Cited by 1 · see citing works

The potential use of anti-codon engineered tRNAs (ACE-tRNAs) to treat nonsense variants causing inherited retinal diseases

Sarma, A. S., & Sharon, D. (2025) · Molecular Aspects of Medicine · doi:10.1016/j.mam.2025.101443 · Cited by 1 · see citing works

Precision Is Not Enough: When Tools Outpace Translation in Ocular Gene Therapy

Elsayed, M. E. A. A., & MacLaren, R. E. (2026) · Genes · doi:10.3390/genes17030283 · Cited by 1 · see citing works

First results of long-term follow-up of children in Russia after gene therapy for hereditary retinal dystrophies associated with biallelic mutations in the RPE65 gene

Neroev, V. V., Катаргина, Л. А., Kharlampidi, M. P., Коголева, Л. В., Zolnikova, I. V., Ilyukhin, P. A., Denisova, E. V., Викторович, М. С., Осипова, Н. А., Kutsev, S. I., et al. (2023) · Russian Ophthalmological Journal · doi:10.21516/2072-0076-2023-16-4-50-62 · Cited by 1 · see citing works

Assessing the impact of single or short-term administration on a therapy’s cost-effectiveness: a hypothetical disease-agnostic model

Klimchak, A. C., Sedita, L. E., Gooch, K. L., & Malone, D. C. (2023) · Journal of Medical Economics · doi:10.1080/13696998.2023.2200102 · Cited by 1 · see citing works

Selective Block of Upregulated Kv1.3 Potassium Channels in ON-Bipolar Cells of the Blind Retina Enhances Optogenetically Restored SignalingPreprint

Kleinlogel, S., Schilardi, G., & Kralik, J. (2023) · Preprints.org · doi:10.20944/preprints202305.0848.v1

Gen- und Zelltherapie 2.023 - Forschung, klinische Anwendung und Gesellschaft

Fehse, B., Schickl, H., Bartfeld, S., & Zenke, M. (2023) · Springer eBooks · doi:10.1007/978-3-662-67908-1

Characterizing the Biopotency of Truncated Transgene Variants in rAAV8 viral vectors: Essential Considerations for Gene Therapy ApplicationsPreprint

Fiedler, C., Lengler, J., Gruber, B., Scheindel, M., Buengener, C., Mittergradnegger, D., Bendik, M., Kraus, B., & Bort, J. A. H. (2024) · Research Square · doi:10.21203/rs.3.rs-4043201/v1

Gene and Cellular Therapies for Retinal Diseases

Neto, P. P. D. S., Silva, L. F., de Queiroz, E. V., Medeiros, J. V. J. D. B., & Teixeira, P. G. (2025) · Brazilian Journal of Implantology and Health Sciences · doi:10.36557/2674-8169.2025v7n1p140-153

Gene Therapy for Inherited Ocular Disorders

Tuszynski, M. H., & Rosenzweig, E. S. (2025) · Springer eBooks · doi:10.1007/978-3-031-89307-0_8

HLA-B27 as a potential target for the cure of axial spondyloarthritis

Cannistrà, C., Timilsina, S., Gershwin, M. E., & Selmi, C. F. (2025) · Current Opinion in Immunology · doi:10.1016/j.coi.2025.102611

Gene Therapy with Voretigene Neparvovec (Luxturna®)

Gerhardt, M. J., & Priglinger, S. G. (2025) · Springer eBooks · doi:10.1007/978-3-031-84204-7_15

Real-world durability challenges with nAMD treatments and the potential promise of gene therapy

Munk, M., Holz, F. G., Chow, D., Sala‐Puigdollers, A., Arias, L., Heier, J., Sakamoto, T., Souied, E. H., Godfrey, J., Douglas, K., et al. (2026) · Current Medical Research and Opinion · doi:10.1080/03007995.2026.2670008

Gene therapy development for retinal diseases

Reichel, F. F., Bellingrath, J.-S., Király, P., & FISCHER, M. D. (2026) · Handbook of clinical neurology · doi:10.1016/b978-0-443-22212-2.00016-1

Intervening in aging and related diseases with gene therapy techniques

Jiang, M., Qu, J., Song, M., & Liu, G. (2026) · Cell Reports Medicine · doi:10.1016/j.xcrm.2026.102870

Mastering the eye: Clinical paradigm shifts from optical restoration to genomic correction

Zhang, X., Li, F., Huang, D., Yu‐Wai‐Man, P., Grzybowski, A. E., Aung, T., Lam, D. S. C., & Weinreb, R. N. (2026) · Progress in Retinal and Eye Research · doi:10.1016/j.preteyeres.2026.101537

How long does gene therapy last? 4-year follow-up of phase 3 voretigene neparvovec trial in RPE65-associated LCA/inherited retinal disease

Drack, A. V., Bennett, J., Russell, S. R., High, K. A., Yu, Z., Tillman, A., Chung, D. C., Reape, K. Z., Ciulla, T. A., & Maguire, A. (2019) · Journal of American Association for Pediatric Ophthalmology and Strabismus · doi:10.1016/j.jaapos.2019.08.018 · Cited by 20 · see citing works

First results of long-term follow-up of children in Russia after gene therapy for hereditary retinal dystrophies associated with biallelic mutations in the RPE65 gene

Neroev, V. V., Катаргина, Л. А., Kharlampidi, M. P., Коголева, Л. В., Zolnikova, I. V., Ilyukhin, P. A., Denisova, E. V., Викторович, М. С., Осипова, Н. А., Kutsev, S. I., et al. (2023) · Russian Ophthalmological Journal · doi:10.21516/2072-0076-2023-16-4-50-62 · Cited by 1 · see citing works

Gene Therapy for Inherited Ocular Disorders

Tuszynski, M. H., & Rosenzweig, E. S. (2025) · Springer eBooks · doi:10.1007/978-3-031-89307-0_8

Sustained Rescue of Rod Function and Probable Non–Cell-Autonomous Rescue of Cones after Gene Therapy

Park, K. S., de Carvalho, J. R. L., & Tsang, S. H. (2019) · Ophthalmology · doi:10.1016/j.ophtha.2019.03.007 · Cited by 1 · see citing works

Voretigene Neparvovec: A Review in RPE65 Mutation-Associated Inherited Retinal Dystrophy

Kang, C., & Scott, L. J. (2020) · Molecular Diagnosis & Therapy · doi:10.1007/s40291-020-00475-6 · Cited by 37 · see citing works