FDA approves hereditary blindness gene therapy

First AAV gene therapy poised for landmark approval

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Adeno-associated virus as a delivery vector for gene therapy of human diseases

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Systemic AAV Micro-dystrophin Gene Therapy for Duchenne Muscular Dystrophy

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Amyotrophic lateral sclerosis: translating genetic discoveries into therapies

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Intein-mediated protein trans-splicing expands adeno-associated virus transfer capacity in the retina

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Immunogenicity of Recombinant Adeno-Associated Virus (AAV) Vectors for Gene Transfer

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Seeing the light after 25 Years of retinal gene therapy

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Ocular gene therapies in clinical practice: viral vectors and nonviral alternatives

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Immune Response Mechanisms against AAV Vectors in Animal Models

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Age-Related Seroprevalence of Antibodies Against AAV-LK03 in a UK Population Cohort

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Genetic testing and diagnosis of inherited retinal diseases

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Adeno-Associated Viral Vectors as a Tool for Large Gene Delivery to the Retina

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Updates on Gene Therapy for Diabetic Retinopathy

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Extracellular vesicles: emerging anti-cancer drugs and advanced functionalization platforms for cancer therapy

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Circumventing the packaging limit of AAV-mediated gene replacement therapy for neurological disorders

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Tumor-Specific Delivery of Immune Checkpoint Inhibitors by Engineered AAV Vectors

Reul, J., Frisch, J., Engeland, C. E., Thalheimer, F. B., Hartmann, J., Ungerechts, G., & Buchholz, C. J. (2019) · Frontiers in Oncology · doi:10.3389/fonc.2019.00052 · Cited by 56 · see citing works

Development of New Strategies Using Extracellular Vesicles Loaded with Exogenous Nucleic Acid

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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

Recombinant adeno-associated virus as a delivery platform for ocular gene therapy: A comprehensive review

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Argininosuccinic aciduria fosters neuronal nitrosative stress reversed by Asl gene transfer

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Therapeutic homology-independent targeted integration in retina and liver

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AAV-Mediated Gene Delivery to the Liver: Overview of Current Technologies and Methods

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Codon-Optimization of Wild-Type Adeno-Associated Virus Capsid Sequences Enhances DNA Family Shuffling while Conserving Functionality

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Exosomes as an Emerging Plasmid Delivery Vehicle for Gene Therapy

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Resolving Neurodevelopmental and Vision Disorders Using Organoid Single-Cell Multi-omics

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Leveraging rAAV bioprocess understanding and next generation bioanalytics development

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Efficacy, pharmacokinetics, and safety in the mouse and primate retina of dual AAV vectors for Usher syndrome type 1B

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Inclusion of a degron reduces levels of undesired inteins after AAV-mediated protein trans-splicing in the retina

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Ocular Gene Therapy: An Overview of Viral Vectors, Immune Responses, and Future Directions

Banou, L., Sarrafpour, S., Teng, C. C., & Liu, J. Y. (2024) · The Yale Journal of Biology and Medicine · doi:10.59249/hwid7537 · Cited by 25 · see citing works

The Landscape of Non-Viral Gene Augmentation Strategies for Inherited Retinal Diseases

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Adeno-associated virus vectors for retinal gene therapy in basic research and clinical studies

Xia, X., & Guo, X. V. (2023) · Frontiers in Medicine · doi:10.3389/fmed.2023.1310050 · Cited by 20 · see citing works

Retinal Stem/Progenitor Cells Derived From Adult Müller Glia for the Treatment of Retinal Degeneration

Too, L. K., & Simunovic, M. P. (2021) · Frontiers in Cell and Developmental Biology · doi:10.3389/fcell.2021.749131 · Cited by 21 · see citing works

A Comparative Analysis of Models for AAV-Mediated Gene Therapy for Inherited Retinal Diseases

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Superior lentiviral vectors designed for BSL-0 environment abolish vector mobilization

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Rollout of high-priced cell and gene therapies forces payer rethink

Senior, M. (2018) · Nature Biotechnology · doi:10.1038/nbt0418-291a · Cited by 14 · see citing works

TPP Combined with DGUC as an Economic and Universal Process for Large-Scale Purification of AAV Vectors

Yu, Z., Zhou, S., Luo, N., Ho, C. Y., Chen, M., & Chen, H. (2019) · Molecular Therapy — Methods & Clinical Development · doi:10.1016/j.omtm.2019.11.009 · Cited by 11 · see citing works

Retinal and Choroidal Neovascularization Antivascular Endothelial Growth Factor Treatments: The Role of Gene Therapy

Toutounchian, S., Ahmadbeigi, N., & Mansouri, V. (2022) · Journal of Ocular Pharmacology and Therapeutics · doi:10.1089/jop.2022.0022 · Cited by 9 · see citing works

Next-generation sequencing-based genetic testing and phenotype correlation in retinitis pigmentosa patients from India

Sen, P., Srikrupa, N. N., Maitra, P., Sundaramurthy, S., Periyasamy, P., Gnanasekaran, H., Bhende, M. P., Khetan, V., Mathavan, S., Bhende, P., et al. (2023) · Indian Journal of Ophthalmology · doi:10.4103/ijo.ijo_2579_22 · Cited by 8 · see citing works

To win at gene therapy, companies pick viruses with production credentials

Sheridan, C. (2019) · Nature Biotechnology · doi:10.1038/nbt0119-5 · Cited by 8 · see citing works

RNA therapeutics to control fibrinolysis: review on applications in biology and medicine

Ferraresso, F., Leung, J., & Kastrup, C. J. (2024) · Journal of Thrombosis and Haemostasis · doi:10.1016/j.jtha.2024.04.006 · Cited by 6 · see citing works

Analysis of the impact of pluronic acid on the thermal stability and infectivity of AAV6.2FF

Thomas, S. P., Spinelli, M. M., Rghei, A. D., Lopes, J. A., Zielinska, N., Mcleod, B. M., Pei, Y.-L., Zhang, W., Thébaud, B., Karimi, K., et al. (2024) · BMC Biotechnology · doi:10.1186/s12896-024-00853-6 · Cited by 7 · see citing works

Tackling visual impairment: emerging avenues in ophthalmology

Lin, F., Su, Y., Zhao, C., Akter, F., Yao, S., Huang, S., Shao, X., & Yao, Y. (2025) · Frontiers in Medicine · doi:10.3389/fmed.2025.1567159 · Cited by 5 · see citing works

Integrating single-cell omics and materials science for uveal melanoma: from mechanistic insights to precision therapeutics

Fu, S., & Li, C. (2025) · Frontiers in Oncology · doi:10.3389/fonc.2025.1661889 · Cited by 4 · see citing works

Transduction of gluconeogenic enzymes prolongs cone photoreceptor survival and function in models of retinitis pigmentosaPreprint

Chinchore, Y., Begaj, T., Guillermeir, C., Steinhauser, M. L., Punzo, C., & Cepko, C. L. (2019) · bioRxiv (Cold Spring Harbor Laboratory) · doi:10.1101/569665 · Cited by 4 · see citing works

Gene therapy and gene editing

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Challenges to Gene Editing Approaches in the Retina

Bonillo, M., Pfromm, J. K., & Fischer, M. D. (2022) · Klinische Monatsblätter für Augenheilkunde · doi:10.1055/a-1757-9810 · Cited by 3 · see citing works

pH-Responsive Conformational-Switching Cationic Fusion Protein for Promoted Plasmid DNA Delivery and Transfection

Wu, J., Tan, T., Chen, J., & Zhang, Y. (2025) · Biomacromolecules · doi:10.1021/acs.biomac.4c01572 · Cited by 2 · see citing works

Technological advances in the diagnosis and management of inherited optic neuropathies

Britton, J. O. T., Yu‐Wai‐Man, P., & Chen, B. S. (2025) · Frontiers in Neurology · doi:10.3389/fneur.2025.1609033 · Cited by 2 · see citing works

A 69 kb Deletion in chr19q13.42 including PRPF31 Gene in a Chinese Family Affected with Autosomal Dominant Retinitis Pigmentosa

Lan, Y., Chen, Y., Qiao, Y., Xu, Q., Zhai, R., Sun, X., Wu, J., & Chen, X. (2022) · Journal of Clinical Medicine · doi:10.3390/jcm11226682 · Cited by 2 · see citing works

A Common Outer Retinal Change in Retinal Degeneration by Optical Coherence Tomography Can Be Used to Assess Outcomes of Gene Therapy

Joe, M. K., Li, W., Hiriyanna, S., Yu, W., Shah, S., Abu-Asab, M. S., Qian, H., & Wu, Z. (2019) · Human Gene Therapy · doi:10.1089/hum.2019.162 · Cited by 2 · see citing works

Strategic lipid nanoparticle design dictates retinal delivery post inner limiting membrane disruption

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Exploring the Genetic Causes of Nonsyndromic Retinal Dystrophies in Qatar

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Exome sequencing identifies novel genetic variants in patients with atypical Non-Syndromic retinitis pigmentosa

Yesharim, L., Hozhabrpour, A., Hosnan, F., Gordiz, A., Hemmati, S., Kazemi, P., Khakpour, G., & Abdi, F. S. (2025) · Molecular Biology Reports · doi:10.1007/s11033-025-11409-y · Cited by 1 · see citing works

From classroom to cleanroom: building a skilled cell and gene therapy workforce

Shokoohmand, A., Tan, E. H. P., Al‐Riyami, A. Z., Koptina, A., Richards, C., Morato-Marques, M., Yoganathan, P., Alrasheed, N., Moir-Meyer, G., Patel, N. M., et al. (2026) · Cytotherapy · doi:10.1016/j.jcyt.2026.102899 · Cited by 1 · see citing works

A Mutation-Agnostic EP2–cAMP–Driven Strategy Restores Ciliogenesis and Retinal Function in CiliopathiesPreprint

de Malglaive, F., Barny, I., Revy, S., Obringer, C., Menguy, L., Machroub, S., Fares‐Taie, L., Cano, E., Goudin, N., Viau, A., et al. (2025) · Research Square · doi:10.21203/rs.3.rs-8000675/v1 · Cited by 1 · see citing works

Optimized AAV capsids robustly transduce airway epithelial cellsPreprint

Cooney, A. L., Chen, Y. H., Lewandowski, B. C., Lamer, S., Boysen, G., Kulhánková, K., Vu, A., Newase, P., Sinn, P. L., Davidson, B. L., et al. (2026) · bioRxiv (Cold Spring Harbor Laboratory) · doi:10.64898/2026.05.10.723853

Streamlined synthetic regulatory cassette for efficient and photoreceptor-enriched retinal gene expression

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Ocular Gene Therapy in China: Technological Framework, Clinical Translation, and Future Perspectives

Zhang, D., He, Z., Lu, F., & Yang, Y. (2026) · Human Gene Therapy · doi:10.1177/10430342261495300

FDA approval of retinal dystrophy drug launches era of ocular gene therapy

Krader, C. G. (2018)

Clinical Perspective: Treating RPE65-Associated Retinal Dystrophy

Maguire, A. M., Bennett, J., Aleman, E. M., Leroy, B. P., & Alemán, T. S. (2020) · Molecular Therapy · doi:10.1016/j.ymthe.2020.11.029 · Cited by 183 · see citing works

Voretigene Neparvovec: An Emerging Gene Therapy for the Treatment of Inherited Blindness

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O Primeiro Tratamento com Luxturna em Portugal: Um Pequeno Passo para a Ciência Mundial, um Salto Gigante para a Oftalmologia Portuguesa

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New Gene Therapy Treatment for Retinitis Pigmentosa

Berezu, T. T. (2020) · NSUWorks (Nova Southeastern University)