{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,8,18]],"date-time":"2026-08-18T04:44:01Z","timestamp":1787028241515,"version":"build-2736575974"},"reference-count":71,"publisher":"MDPI AG","issue":"7","license":[{"start":{"date-parts":[[2025,7,21]],"date-time":"2025-07-21T00:00:00Z","timestamp":1753056000000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"funder":[{"name":"Internal funding from the Research Department, King Khaled Eye Specialist Hospital, Riyadh and the Department of Surgery, Faculty of Medicine, University of Tabuk, Tabuk supported this work.","award":["N\/A"],"award-info":[{"award-number":["N\/A"]}]}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Genes"],"abstract":"<jats:p>Gene therapy has emerged as a promising treatment for several eye diseases since it may restore vision and stop blindness. Many eye diseases, including retinitis pigmentosa and macular degeneration, have historically been rather difficult to treat and usually cause permanent vision loss. However, thanks to advances in gene therapy, many disorders can now be effectively targeted and genetically changed, providing a safer, more direct, maybe even curative approach. By introducing, altering, or repairing specific genes inside the eye, gene therapy seeks to fix the defective genes causing these disorders, thereby improving general eye health and visual ability. Voretigene neparvovec is one FDA- and EMA-approved treatment for RPE65 mutations. Retinitis pigmentosa, age-related macular degeneration, X-linked retinoschisis, choroideremia, and Stargardt disease are among the several eye disorders still under clinical trials, and experimental treatment is in progress. As research on gene therapy develops, it opens the path for groundbreaking treatments that could fundamentally change the ophthalmic care scene.<\/jats:p>","DOI":"10.3390\/genes16070847","type":"journal-article","created":{"date-parts":[[2025,7,21]],"date-time":"2025-07-21T10:35:31Z","timestamp":1753094131000},"page":"847","update-policy":"https:\/\/doi.org\/10.3390\/mdpi_crossmark_policy","source":"Crossref","is-referenced-by-count":7,"title":["Advances in Precision Therapeutics and Gene Therapy Applications for Retinal Diseases: Impact and Future Directions"],"prefix":"10.3390","volume":"16","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-2355-4036","authenticated-orcid":false,"given":"Mariam M.","family":"AlEissa","sequence":"first","affiliation":[{"name":"Research Department, King Khaled Eye Specialist Hospital, Riyadh 11462, Saudi Arabia"},{"name":"College of Medicine, Alfaisal University, Riyadh 11533, Saudi Arabia"},{"name":"Public Health Laboratory, Public Health Authority, Riyadh 12382, Saudi Arabia"},{"name":"The Computational Sciences Department at the Centre for Genomic Medicine (CGM), King Faisal Specialist Hospital & Research Centre, Riyadh 11211, Saudi Arabia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Abrar A.","family":"Alhawsawi","sequence":"additional","affiliation":[{"name":"Division of Ophthalmology, Department of Surgery, College of Medicine, University of Jeddah, Jeddah 23218, Saudi Arabia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0009-0001-6714-4143","authenticated-orcid":false,"given":"Raghad","family":"Alonazi","sequence":"additional","affiliation":[{"name":"College of Medicine, Alfaisal University, Riyadh 11533, Saudi Arabia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Enas","family":"Magharbil","sequence":"additional","affiliation":[{"name":"Retina and Uveitis Department, Jeddah Eye Hospital, Jeddah 23454, Saudi Arabia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-0664-554X","authenticated-orcid":false,"given":"Abeer","family":"Aljahdali","sequence":"additional","affiliation":[{"name":"Ophthalmology Department, King Abdulaziz University, Jeddah 21589, Saudi Arabia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Hani B.","family":"AlBalawi","sequence":"additional","affiliation":[{"name":"Division of Ophthalmology, Department of Surgery, Faculty of Medicine, University of Tabuk, Tabuk 47311, Saudi Arabia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0009-0008-8860-543X","authenticated-orcid":false,"given":"Naif M.","family":"Alali","sequence":"additional","affiliation":[{"name":"Division of Ophthalmology, Department of Surgery, Faculty of Medicine, University of Tabuk, Tabuk 47311, Saudi Arabia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0009-0004-2746-0784","authenticated-orcid":false,"given":"Syed","family":"Hameed","sequence":"additional","affiliation":[{"name":"Research Department, King Khaled Eye Specialist Hospital, Riyadh 11462, Saudi Arabia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Khaled K.","family":"Abu-Amero","sequence":"additional","affiliation":[{"name":"Research Department, King Khaled Eye Specialist Hospital, Riyadh 11462, Saudi Arabia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Moustafa S.","family":"Magliyah","sequence":"additional","affiliation":[{"name":"Research Department, King Khaled Eye Specialist Hospital, Riyadh 11462, Saudi Arabia"},{"name":"Vitreoretinal Division, King Khaled Eye Specialist Hospital, Riyadh 11462, Saudi Arabia"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"1968","published-online":{"date-parts":[[2025,7,21]]},"reference":[{"key":"ref_1","doi-asserted-by":"crossref","unstructured":"Nguyen, X.-T.-A., Moekotte, L., Plomp, A.S., Bergen, A.A., Van Genderen, M.M., and Boon, C.J.F. (2023). Retinitis Pigmentosa: Current Clinical Management and Emerging Therapies. IJMS, 24.","DOI":"10.3390\/ijms24087481"},{"key":"ref_2","doi-asserted-by":"crossref","first-page":"575","DOI":"10.1007\/s40291-024-00722-0","article-title":"Gene Therapies in Clinical Development to Treat Retinal Disorders","volume":"28","author":"McClements","year":"2024","journal-title":"Mol. Diagn. Ther."},{"key":"ref_3","doi-asserted-by":"crossref","first-page":"849","DOI":"10.1016\/S0140-6736(17)31868-8","article-title":"Efficacy and Safety of Voretigene Neparvovec (AAV2-hRPE65v2) in Patients with RPE65-Mediated Inherited Retinal Dystrophy: A Randomised, Controlled, Open-Label, Phase 3 Trial","volume":"390","author":"Russell","year":"2017","journal-title":"Lancet"},{"key":"ref_4","doi-asserted-by":"crossref","first-page":"281","DOI":"10.1136\/bjo-2024-326221","article-title":"12-Month Outcomes after Voretigene Neparvovec Gene Therapy in Paediatric Patients with RPE65-Mediated Inherited Retinal Dystrophy","volume":"109","author":"Daruich","year":"2025","journal-title":"Br. J. Ophthalmol."},{"key":"ref_5","doi-asserted-by":"crossref","first-page":"381","DOI":"10.17762\/ijritcc.v11i7.9421","article-title":"Emerging Therapies in Retinal Diseases: From Gene Therapy to Stem Cell Interventions","volume":"11","author":"Gadre","year":"2023","journal-title":"IJRITCC"},{"key":"ref_6","doi-asserted-by":"crossref","unstructured":"Mahaling, B., Low, S.W.Y., Ch, S., Addi, U.R., Ahmad, B., Connor, T.B., Mohan, R.R., Biswas, S., and Chaurasia, S.S. (2023). Next-Generation Nanomedicine Approaches for the Management of Retinal Diseases. Pharmaceutics, 15.","DOI":"10.3390\/pharmaceutics15072005"},{"key":"ref_7","doi-asserted-by":"crossref","first-page":"327","DOI":"10.1007\/s00439-016-1637-y","article-title":"Treatment of Retinitis Pigmentosa Due to MERTK Mutations by Ocular Subretinal Injection of Adeno-Associated Virus Gene Vector: Results of a Phase I Trial","volume":"135","author":"Ghazi","year":"2016","journal-title":"Hum. Genet."},{"key":"ref_8","doi-asserted-by":"crossref","first-page":"354","DOI":"10.1038\/s41591-020-0763-1","article-title":"Initial Results from a First-in-Human Gene Therapy Trial on X-Linked Retinitis Pigmentosa Caused by Mutations in RPGR","volume":"26","author":"Xue","year":"2020","journal-title":"Nat. Med."},{"key":"ref_9","doi-asserted-by":"crossref","first-page":"1129","DOI":"10.1016\/S0140-6736(13)62117-0","article-title":"Retinal Gene Therapy in Patients with Choroideremia: Initial Findings from a Phase 1\/2 Clinical Trial","volume":"383","author":"MacLaren","year":"2014","journal-title":"Lancet"},{"key":"ref_10","doi-asserted-by":"crossref","first-page":"1996","DOI":"10.1056\/NEJMc1509501","article-title":"Visual Acuity after Retinal Gene Therapy for Choroideremia","volume":"374","author":"Edwards","year":"2016","journal-title":"N. Engl. J. Med."},{"key":"ref_11","doi-asserted-by":"crossref","first-page":"130","DOI":"10.1016\/j.ajo.2018.06.011","article-title":"Two-Year Results After AAV2-Mediated Gene Therapy for Choroideremia: The Alberta Experience","volume":"193","author":"Dimopoulos","year":"2018","journal-title":"Am. J. Ophthalmol."},{"key":"ref_12","doi-asserted-by":"crossref","first-page":"2464","DOI":"10.1038\/s41591-023-02520-3","article-title":"Subretinal Timrepigene Emparvovec in Adult Men with Choroideremia: A Randomized Phase 3 Trial","volume":"29","author":"MacLaren","year":"2023","journal-title":"Nat. Med."},{"key":"ref_13","doi-asserted-by":"crossref","first-page":"823","DOI":"10.1016\/j.omtn.2022.08.026","article-title":"Effective Gene Therapy of Stargardt Disease with PEG-ECO\/pGRK1-ABCA4-S\/MAR Nanoparticles","volume":"29","author":"Sun","year":"2022","journal-title":"Mol. Ther.\u2014Nucleic Acids"},{"key":"ref_14","first-page":"343","article-title":"Uber Das Zusammenvorkommen von Veraderungen Der Retina Und Chorioidea","volume":"37","author":"HAAS","year":"1898","journal-title":"Arch. Augenheilked"},{"key":"ref_15","doi-asserted-by":"crossref","first-page":"191","DOI":"10.1016\/j.ophtha.2021.09.021","article-title":"X-Linked Retinoschisis","volume":"129","author":"Hahn","year":"2022","journal-title":"Ophthalmology"},{"key":"ref_16","doi-asserted-by":"crossref","first-page":"585","DOI":"10.1038\/gt.2014.31","article-title":"Retinoschisin Gene Therapy in Photoreceptors, M\u00fcller Glia or All Retinal Cells in the Rs1h\u2212\/\u2212 Mouse","volume":"21","author":"Byrne","year":"2014","journal-title":"Gene Ther."},{"key":"ref_17","doi-asserted-by":"crossref","first-page":"34","DOI":"10.1186\/s40662-016-0063-5","article-title":"Epidemiology of Age-Related Macular Degeneration (AMD): Associations with Cardiovascular Disease Phenotypes and Lipid Factors","volume":"3","author":"Pennington","year":"2016","journal-title":"Eye Vis."},{"key":"ref_18","doi-asserted-by":"crossref","first-page":"e264","DOI":"10.1038\/mtna.2015.37","article-title":"Off-Target Effects in CRISPR\/Cas9-Mediated Genome Engineering","volume":"4","author":"Zhang","year":"2015","journal-title":"Mol. Ther.\u2014Nucleic Acids"},{"key":"ref_19","doi-asserted-by":"crossref","first-page":"278","DOI":"10.1016\/j.ajhg.2020.06.011","article-title":"Human iPSC Modeling Reveals Mutation-Specific Responses to Gene Therapy in a Genotypically Diverse Dominant Maculopathy","volume":"107","author":"Sinha","year":"2020","journal-title":"Am. J. Hum. Genet."},{"key":"ref_20","doi-asserted-by":"crossref","first-page":"1434","DOI":"10.1016\/S0140-6736(23)01520-9","article-title":"Pegcetacoplan for the Treatment of Geographic Atrophy Secondary to Age-Related Macular Degeneration (OAKS and DERBY): Two Multicentre, Randomised, Double-Masked, Sham-Controlled, Phase 3 Trials","volume":"402","author":"Heier","year":"2023","journal-title":"Lancet"},{"key":"ref_21","doi-asserted-by":"crossref","first-page":"1449","DOI":"10.1016\/S0140-6736(23)01583-0","article-title":"Efficacy and Safety of Avacincaptad Pegol in Patients with Geographic Atrophy (GATHER2): 12-Month Results from a Randomised, Double-Masked, Phase 3 Trial","volume":"402","author":"Khanani","year":"2023","journal-title":"Lancet"},{"key":"ref_22","doi-asserted-by":"crossref","first-page":"1076","DOI":"10.1016\/j.ymthe.2017.03.008","article-title":"Taking Stock of Retinal Gene Therapy: Looking Back and Moving Forward","volume":"25","author":"Bennett","year":"2017","journal-title":"Mol. Ther."},{"key":"ref_23","doi-asserted-by":"crossref","first-page":"2545","DOI":"10.1016\/S0042-6989(98)00308-3","article-title":"Persistent Transgene Product in Retina, Optic Nerve and Brain after Intraocular Injection of rAAV","volume":"39","author":"Dudus","year":"1999","journal-title":"Vis. Res."},{"key":"ref_24","doi-asserted-by":"crossref","first-page":"141","DOI":"10.1006\/mcne.2002.1168","article-title":"Intravitreal Injection of Adeno-Associated Viral Vectors Results in the Transduction of Different Types of Retinal Neurons in Neonatal and Adult Rats: A Comparison with Lentiviral Vectors","volume":"21","author":"Harvey","year":"2002","journal-title":"Mol. Cell. Neurosci."},{"key":"ref_25","doi-asserted-by":"crossref","first-page":"e13392","DOI":"10.15252\/emmm.202013392","article-title":"Novel AAV Capsids for Intravitreal Gene Therapy of Photoreceptor Disorders","volume":"13","author":"Pavlou","year":"2021","journal-title":"EMBO Mol. Med."},{"key":"ref_26","doi-asserted-by":"crossref","first-page":"107","DOI":"10.1016\/j.preteyeres.2017.10.004","article-title":"Molecular Genetics and Emerging Therapies for Retinitis Pigmentosa: Basic Research and Clinical Perspectives","volume":"63","author":"Dias","year":"2018","journal-title":"Prog. Retin. Eye Res."},{"key":"ref_27","doi-asserted-by":"crossref","unstructured":"Liu, W., Liu, S., Li, P., and Yao, K. (2022). Retinitis Pigmentosa: Progress in Molecular Pathology and Biotherapeutical Strategies. IJMS, 23.","DOI":"10.3390\/ijms23094883"},{"key":"ref_28","doi-asserted-by":"crossref","first-page":"81","DOI":"10.1038\/s41434-021-00258-6","article-title":"Restoration of RPGR Expression in Vivo Using CRISPR\/Cas9 Gene Editing","volume":"29","author":"Gumerson","year":"2022","journal-title":"Gene Ther."},{"key":"ref_29","doi-asserted-by":"crossref","first-page":"7695","DOI":"10.1038\/s41467-022-35361-8","article-title":"Gene Augmentation Prevents Retinal Degeneration in a CRISPR\/Cas9-Based Mouse Model of PRPF31 Retinitis Pigmentosa","volume":"13","author":"Xi","year":"2022","journal-title":"Nat. Commun."},{"key":"ref_30","doi-asserted-by":"crossref","first-page":"108","DOI":"10.1016\/j.preteyeres.2014.08.001","article-title":"Vector Platforms for Gene Therapy of Inherited Retinopathies","volume":"43","author":"Trapani","year":"2014","journal-title":"Prog. Retin. Eye Res."},{"key":"ref_31","doi-asserted-by":"crossref","first-page":"120ra15","DOI":"10.1126\/scitranslmed.3002865","article-title":"AAV2 Gene Therapy Readministration in Three Adults with Congenital Blindness","volume":"4","author":"Bennett","year":"2012","journal-title":"Sci. Transl. Med."},{"key":"ref_32","doi-asserted-by":"crossref","first-page":"331","DOI":"10.1016\/j.ejpb.2014.12.022","article-title":"Treatment of Ocular Disorders by Gene Therapy","volume":"95","author":"Apaolaza","year":"2015","journal-title":"Eur. J. Pharm. Biopharm."},{"key":"ref_33","doi-asserted-by":"crossref","first-page":"1597","DOI":"10.1016\/S0140-6736(09)61836-5","article-title":"Age-Dependent Effects of RPE65 Gene Therapy for Leber\u2019s Congenital Amaurosis: A Phase 1 Dose-Escalation Trial","volume":"374","author":"Maguire","year":"2009","journal-title":"Lancet"},{"key":"ref_34","doi-asserted-by":"crossref","first-page":"831","DOI":"10.1136\/bjophthalmol-2020-318286","article-title":"Spatial and Temporal Resolution of the Photoreceptors Rescue Dynamics after Treatment with Voretigene Neparvovec","volume":"106","author":"Stingl","year":"2022","journal-title":"Br. J. Ophthalmol."},{"key":"ref_35","doi-asserted-by":"crossref","first-page":"273","DOI":"10.1016\/j.oret.2021.11.005","article-title":"Visual Acuity, Retinal Morphology, and Patients\u2019 Perceptions after Voretigene Neparovec-Rzyl Therapy for RPE65-Associated Retinal Disease","volume":"6","author":"Sengillo","year":"2022","journal-title":"Ophthalmol. Retin."},{"key":"ref_36","doi-asserted-by":"crossref","first-page":"161","DOI":"10.1016\/j.ophtha.2023.09.006","article-title":"Single Center Experience with Voretigene Neparvovec Gene Augmentation Therapy in RPE65 Mutation\u2013Associated Inherited Retinal Degeneration in a Clinical Setting","volume":"131","author":"Lorenz","year":"2024","journal-title":"Ophthalmology"},{"key":"ref_37","doi-asserted-by":"crossref","first-page":"17","DOI":"10.1167\/tvst.12.4.17","article-title":"Improved Rod Sensitivity as Assessed by Two-Color Dark-Adapted Perimetry in Patients with RPE65-Related Retinopathy Treated With Voretigene Neparvovec-Rzyl","volume":"12","author":"Ku","year":"2023","journal-title":"Trans. Vis. Sci. Tech."},{"key":"ref_38","doi-asserted-by":"crossref","first-page":"58","DOI":"10.1016\/j.oret.2021.03.016","article-title":"Perifoveal Chorioretinal Atrophy after Subretinal Voretigene Neparvovec-Rzyl for RPE65-Mediated Leber Congenital Amaurosis","volume":"6","author":"Gange","year":"2022","journal-title":"Ophthalmol. Retin."},{"key":"ref_39","doi-asserted-by":"crossref","unstructured":"Fischer, M.D., Simonelli, F., Sahni, J., Holz, F.G., Maier, R., Fasser, C., Suhner, A., Stiehl, D.P., Chen, B., and Audo, I. (2024). Real-World Safety and Effectiveness of Voretigene Neparvovec: Results up to 2 Years from the Prospective, Registry-Based PERCEIVE Study. Biomolecules, 14.","DOI":"10.3390\/biom14010122"},{"key":"ref_40","doi-asserted-by":"crossref","first-page":"1543","DOI":"10.1007\/s00417-021-05508-2","article-title":"Real-World Outcomes of Voretigene Neparvovec Treatment in Pediatric Patients with RPE65-Associated Leber Congenital Amaurosis","volume":"260","author":"Deng","year":"2022","journal-title":"Graefes Arch. Clin. Exp. Ophthalmol."},{"key":"ref_41","doi-asserted-by":"crossref","first-page":"1460","DOI":"10.1016\/j.ophtha.2021.03.031","article-title":"Durability of Voretigene Neparvovec for Biallelic RPE65-Mediated Inherited Retinal Disease","volume":"128","author":"Maguire","year":"2021","journal-title":"Ophthalmology"},{"key":"ref_42","first-page":"47","article-title":"Long-Term Experience with Gene Augmentation Therapy in Patients with Inherited Retinal Disease Associated with Biallelic Mutations in RPE65","volume":"37","author":"Lorenz","year":"2025","journal-title":"Med. Genet."},{"key":"ref_43","doi-asserted-by":"crossref","first-page":"661","DOI":"10.1016\/S0140-6736(16)30371-3","article-title":"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","volume":"388","author":"Bennett","year":"2016","journal-title":"Lancet"},{"key":"ref_44","doi-asserted-by":"crossref","first-page":"42","DOI":"10.1016\/j.oret.2023.08.017","article-title":"Classification and Growth Rate of Chorioretinal Atrophy after Voretigene Neparvovec-Rzyl for RPE65-Mediated Retinal Degeneration","volume":"8","author":"Bommakanti","year":"2024","journal-title":"Ophthalmol. Retin."},{"key":"ref_45","doi-asserted-by":"crossref","first-page":"1287","DOI":"10.1016\/j.ophtha.2022.06.018","article-title":"Inflammation after Voretigene Neparvovec Administration in Patients with RPE65-Related Retinal Dystrophy","volume":"129","author":"Kessel","year":"2022","journal-title":"Ophthalmology"},{"key":"ref_46","doi-asserted-by":"crossref","first-page":"299","DOI":"10.1136\/bjo-2022-321488","article-title":"Subretinal Deposits in Young Patients Treated with Voretigene Neparvovec-Rzyl for RPE65-Mediated Retinal Dystrophy","volume":"107","author":"Lopez","year":"2023","journal-title":"Br. J. Ophthalmol."},{"key":"ref_47","doi-asserted-by":"crossref","first-page":"1331","DOI":"10.1136\/bjophthalmol-2021-321023","article-title":"Development of Retinal Atrophy after Subretinal Gene Therapy with Voretigene Neparvovec","volume":"107","author":"Reichel","year":"2023","journal-title":"Br. J. Ophthalmol."},{"key":"ref_48","doi-asserted-by":"crossref","first-page":"768","DOI":"10.1007\/s12033-021-00345-4","article-title":"CRISPR-Based Genome Editing as a New Therapeutic Tool in Retinal Diseases","volume":"63","author":"Rasoulinejad","year":"2021","journal-title":"Mol. Biotechnol."},{"key":"ref_49","doi-asserted-by":"crossref","first-page":"229","DOI":"10.1038\/s41591-018-0327-9","article-title":"Development of a Gene-Editing Approach to Restore Vision Loss in Leber Congenital Amaurosis Type 10","volume":"25","author":"Maeder","year":"2019","journal-title":"Nat. Med."},{"key":"ref_50","doi-asserted-by":"crossref","first-page":"1972","DOI":"10.1056\/NEJMoa2309915","article-title":"Gene Editing for CEP290-Associated Retinal Degeneration","volume":"390","author":"Pierce","year":"2024","journal-title":"N. Engl. J. Med."},{"key":"ref_51","doi-asserted-by":"crossref","first-page":"1753","DOI":"10.2147\/OPTH.S364200","article-title":"Ocular Gene Therapy: A Literature Review with Special Focus on Immune and Inflammatory Responses","volume":"16","author":"Ghoraba","year":"2022","journal-title":"OPTH"},{"key":"ref_52","doi-asserted-by":"crossref","first-page":"147","DOI":"10.1097\/ICU.0000000000000660","article-title":"Gene Therapy beyond Luxturna: A New Horizon of the Treatment for Inherited Retinal Disease","volume":"31","author":"Prado","year":"2020","journal-title":"Curr. Opin. Ophthalmol."},{"key":"ref_53","doi-asserted-by":"crossref","first-page":"161","DOI":"10.1016\/j.jval.2018.09.2841","article-title":"Cost Utility of Voretigene Neparvovec for Biallelic RPE65-Mediated Inherited Retinal Disease","volume":"22","author":"Zimmermann","year":"2019","journal-title":"Value Health"},{"key":"ref_54","doi-asserted-by":"crossref","first-page":"249","DOI":"10.3109\/13816810.2015.1121501","article-title":"Ethical Considerations in Gene Therapy","volume":"37","author":"Levin","year":"2016","journal-title":"Ophthalmic Genet."},{"key":"ref_55","doi-asserted-by":"crossref","first-page":"32","DOI":"10.1631\/jzus.B1800624","article-title":"Experiments That Led to the First Gene-Edited Babies: The Ethical Failings and the Urgent Need for Better Governance","volume":"20","author":"Li","year":"2019","journal-title":"J. Zhejiang Univ. Sci. B"},{"key":"ref_56","first-page":"1121","article-title":"After He Jiankui\u2019s Case: Chinese Legislative Modifications in Human Embryo Gene Editing","volume":"33","author":"Chen","year":"2022","journal-title":"Hum. Gene Ther."},{"key":"ref_57","doi-asserted-by":"crossref","unstructured":"Olaghere, J., Williams, D.A., Farrar, J., B\u00fcning, H., Calhoun, C., Ho, T., Inamdar, M.S., Liu, D., Makani, J., and Nyarko, K. (2025). Scientific Advancements in Gene Therapies: Opportunities for Global Regulatory Convergence. Biomedicines, 13.","DOI":"10.3390\/biomedicines13030758"},{"key":"ref_58","doi-asserted-by":"crossref","first-page":"317","DOI":"10.1007\/s40259-017-0234-5","article-title":"Adeno-Associated Virus (AAV) as a Vector for Gene Therapy","volume":"31","author":"Naso","year":"2017","journal-title":"BioDrugs"},{"key":"ref_59","doi-asserted-by":"crossref","first-page":"375","DOI":"10.1002\/jgm.1126","article-title":"Novel AAV Serotypes for Improved Ocular Gene Transfer","volume":"10","author":"Lebherz","year":"2008","journal-title":"J. Gene Med."},{"key":"ref_60","doi-asserted-by":"crossref","first-page":"29","DOI":"10.1007\/s11095-018-2554-7","article-title":"Pharmaceutical Development of AAV-Based Gene Therapy Products for the Eye","volume":"36","author":"Rodrigues","year":"2019","journal-title":"Pharm. Res."},{"key":"ref_61","doi-asserted-by":"crossref","first-page":"1685","DOI":"10.1016\/j.drudis.2019.05.038","article-title":"Ocular Gene Therapies in Clinical Practice: Viral Vectors and Nonviral Alternatives","volume":"24","author":"Bordet","year":"2019","journal-title":"Drug Discov. Today"},{"key":"ref_62","doi-asserted-by":"crossref","unstructured":"Aldhamen, Y.A., Seregin, S.S., and Amalfitano, A. (2011). Immune Recognition of Gene Transfer Vectors: Focus on Adenovirus as a Paradigm. Front. Immunol., 2.","DOI":"10.3389\/fimmu.2011.00040"},{"key":"ref_63","doi-asserted-by":"crossref","first-page":"123","DOI":"10.1038\/nrd1955","article-title":"Pegaptanib, a Targeted Anti-VEGF Aptamer for Ocular Vascular Disease","volume":"5","author":"Ng","year":"2006","journal-title":"Nat. Rev. Drug Discov."},{"key":"ref_64","doi-asserted-by":"crossref","unstructured":"Kharisova, C.B., Kitaeva, K.V., Solovyeva, V.V., Sufianov, A.A., Sufianova, G.Z., Akhmetshin, R.F., Bulgar, S.N., and Rizvanov, A.A. (2025). Looking to the Future of Viral Vectors in Ocular Gene Therapy: Clinical Review. Biomedicines, 13.","DOI":"10.3390\/biomedicines13020365"},{"key":"ref_65","doi-asserted-by":"crossref","first-page":"1224","DOI":"10.1016\/j.addr.2006.07.023","article-title":"Non-Viral Ocular Gene Therapy: Potential Ocular Therapeutic Avenues","volume":"58","author":"Bloquel","year":"2006","journal-title":"Adv. Drug Deliv. Rev."},{"key":"ref_66","doi-asserted-by":"crossref","first-page":"761","DOI":"10.1038\/s41434-023-00419-9","article-title":"The Estimated Annual Financial Impact of Gene Therapy in the United States","volume":"30","author":"Wong","year":"2023","journal-title":"Gene Ther."},{"key":"ref_67","doi-asserted-by":"crossref","first-page":"949","DOI":"10.1016\/j.drudis.2019.01.019","article-title":"Luxturna: FDA Documents Reveal the Value of a Costly Gene Therapy","volume":"24","author":"Darrow","year":"2019","journal-title":"Drug Discov. Today"},{"key":"ref_68","doi-asserted-by":"crossref","first-page":"90","DOI":"10.1016\/j.ajo.2021.08.009","article-title":"Challenges of Cost-Effectiveness Analyses of Novel Therapeutics for Inherited Retinal Diseases","volume":"235","author":"Jayasundera","year":"2022","journal-title":"Am. J. Ophthalmol."},{"key":"ref_69","doi-asserted-by":"crossref","first-page":"383","DOI":"10.1007\/s40273-021-01003-y","article-title":"Methodological Challenges in the Economic Evaluation of a Gene Therapy for RPE65-Mediated Inherited Retinal Disease: The Value of Vision","volume":"39","author":"Huygens","year":"2021","journal-title":"PharmacoEconomics"},{"key":"ref_70","doi-asserted-by":"crossref","first-page":"1115","DOI":"10.1001\/jamaophthalmol.2019.2512","article-title":"Cost-Effectiveness of Voretigene Neparvovec-Rzyl vs Standard Care for RPE65-Mediated Inherited Retinal Disease","volume":"137","author":"Johnson","year":"2019","journal-title":"JAMA Ophthalmol."},{"key":"ref_71","doi-asserted-by":"crossref","first-page":"747","DOI":"10.1038\/s41434-023-00392-3","article-title":"Towards Affordable CRISPR Genomic Therapies: A Task Force Convened by the Innovative Genomics Institute","volume":"30","author":"Witkowsky","year":"2023","journal-title":"Gene Ther."}],"container-title":["Genes"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/www.mdpi.com\/2073-4425\/16\/7\/847\/pdf","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2025,10,9]],"date-time":"2025-10-09T18:13:04Z","timestamp":1760033584000},"score":1,"resource":{"primary":{"URL":"https:\/\/www.mdpi.com\/2073-4425\/16\/7\/847"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2025,7,21]]},"references-count":71,"journal-issue":{"issue":"7","published-online":{"date-parts":[[2025,7]]}},"alternative-id":["genes16070847"],"URL":"https:\/\/doi.org\/10.3390\/genes16070847","relation":{},"ISSN":["2073-4425"],"issn-type":[{"value":"2073-4425","type":"electronic"}],"subject":[],"published":{"date-parts":[[2025,7,21]]}}}