Nanoparticle-based technologies for retinal gene therapy

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Direct Gene Transfer into Mouse Muscle in Vivo

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Mechanisms of endocytosis

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To exploit the tumor microenvironment: Passive and active tumor targeting of nanocarriers for anti-cancer drug delivery

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Gold nanoparticles in delivery applications☆

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Effect of Surface Properties on Nanoparticle–Cell Interactions

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Molecular mechanism and physiological functions of clathrin-mediated endocytosis

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

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Designing dendrimers for biological applications

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Effect of gene therapy on visual function in Leber’s congenital amaurosis

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Toxicity of Gold Nanoparticles Functionalized with Cationic and Anionic Side Chains

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Nanocarriers’ entry into the cell: relevance to drug delivery

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CTCF: Master Weaver of the Genome

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Albumin-based nanoparticles as potential controlled release drug delivery systems

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A molecular ruler based on plasmon coupling of single gold and silver nanoparticles

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Sterilization, toxicity, biocompatibility and clinical applications of polylactic acid/ polyglycolic acid copolymers

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The multiple faces of caveolae

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Structure of DNA-Cationic Liposome Complexes: DNA Intercalation in Multilamellar Membranes in Distinct Interhelical Packing Regimes

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Cellular and Molecular Barriers to Gene Transfer by a Cationic Lipid

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Chloride Accumulation and Swelling in Endosomes Enhances DNA Transfer by Polyamine-DNA Polyplexes

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Rapid endo‐lysosomal escape of poly(DL‐lactide‐ co glycolide) nanoparticles: implications for drug and gene delivery

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Nano/micro technologies for delivering macromolecular therapeutics using poly(D,L-lactide-co-glycolide) and its derivatives

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Effect of genome size on AAV vector packaging

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Efficient transfer of genetic material into mammalian cells using Starburst polyamidoamine dendrimers.

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Nanomedicine(s) under the Microscope

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Photothermal Imaging of Nanometer-Sized Metal Particles Among Scatterers

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Chitosan-based formulations for delivery of DNA and siRNA

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Role of clathrin- and caveolae-mediated endocytosis in gene transfer mediated by lipo- and polyplexes

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Understanding the Role of Surface Charges in Cellular Adsorption versus Internalization by Selectively Removing Gold Nanoparticles on the Cell Surface with a I 2 /KI Etchant

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Leber congenital amaurosis: Genes, proteins and disease mechanisms

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Cationic Polystyrene Nanosphere Toxicity Depends on Cell-Specific Endocytic and Mitochondrial Injury Pathways

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Human gene therapy for RPE65 isomerase deficiency activates the retinoid cycle of vision but with slow rod kinetics

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Gelatin-based nanoparticles as drug and gene delivery systems: Reviewing three decades of research

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Retinitis Pigmentosa: Genes and Disease Mechanisms

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Transferrin- and transferrin-receptor-antibody-modified nanoparticles enable drug delivery across the blood–brain barrier (BBB)

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Conjugation to gold nanoparticles enhances polyethylenimine's transfer of plasmid DNA into mammalian cells

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Systematic Comparison of Constitutive Promoters and the Doxycycline-Inducible Promoter

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Mutations in the RPE65 gene in patients with autosomal recessive retinitis pigmentosa or Leber congenital amaurosis

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Targeting receptor-mediated endocytotic pathways with nanoparticles: Rationale and advances

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Minicircle DNA vectors devoid of bacterial DNA result in persistent and high-level transgene expression in vivo

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

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Efficient Gene Delivery Vectors by Tuning the Surface Charge Density of Amino Acid-Functionalized Gold Nanoparticles

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Phagocytosis of Retinal Rod and Cone Photoreceptors

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In vivo evaluation of the biodistribution and safety of PLGA nanoparticles as drug delivery systems

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Directional conjugation of antibodies to nanoparticles for synthesis of multiplexed optical contrast agents with both delivery and targeting moieties

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Nanoscopic structure of DNA condensed for gene delivery

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Biological Significance of Unwinding Capability of Nuclear Matrix-Associating DNAs

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Loss of transcriptional activity of a transgene is accompanied by DNA methylation and histone deacetylation and is prevented by insulatorsPreprint

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Enhanced transfection efficiency of PAMAM dendrimer by surface modification with l-arginine

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Phagocytosis of rod outer segments by retinal pigment epithelial cells requires αvβ5 integrin for binding but not for internalization

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Lipid Rafts, Caveolae, and Their Endocytosis

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Ocular immune privilege: the eye takes a dim but practical view of immunity and inflammation

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Transcriptional silencing is associated with extensive methylation of the CMV promoter following adenoviral gene delivery to muscle

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Gene therapy with brain-derived neurotrophic factor as a protection: Retinal ganglion cells in a rat glaucoma model

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Hyaluronic acid based self-assembling nanosystems for CD44 target mediated siRNA delivery to solid tumors

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Ocular Drug Delivery Targeting the Retina and Retinal Pigment Epithelium Using Polylactide Nanoparticles

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Lipoplex-mediated Transfection of Mammalian Cells Occurs through the Cholesterol-dependent Clathrin-mediated Pathway of Endocytosis

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Three-Year Follow-up after Unilateral Subretinal Delivery of Adeno-Associated Virus in Patients with Leber Congenital Amaurosis Type 2

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The use of PAMAM dendrimers in the efficient transfer of genetic material into cells

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Human <i>RPE65</i> Gene Therapy for Leber Congenital Amaurosis: Persistence of Early Visual Improvements and Safety at 1 Year

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Efficient gene delivery targeted to the brain using a transferrin‐conjugated polyethyleneglycol‐modified polyamidoamine dendrimer

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The Genetics of Age-Related Macular Degeneration: A Review of Progress to Date

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Transferrin-Mediated Gold Nanoparticle Cellular Uptake

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Nucleolin-targeting liposomes guided by aptamer AS1411 for the delivery of siRNA for the treatment of malignant melanomas

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Import of Plasmid DNA into the Nucleus Is Sequence Specific

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Enhanced Expression of Transgenes from Adeno-Associated Virus Vectors with the Woodchuck Hepatitis Virus Posttranscriptional Regulatory Element: Implications for Gene Therapy

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PEGylated Gold Nanoparticles Conjugated to Monoclonal F19 Antibodies as Targeted Labeling Agents for Human Pancreatic Carcinoma Tissue

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Effective delivery of large genes to the retina by dual AAV vectors

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Solid lipid nanoparticles for ocular drug delivery

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Novel adeno-associated virus serotypes efficiently transduce murine photoreceptors

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Essential role for MFG-E8 as ligand for αvβ5 integrin in diurnal retinal phagocytosis

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A Columnar Phase of Dendritic Lipid−Based Cationic Liposome−DNA Complexes for Gene Delivery: Hexagonally Ordered Cylindrical Micelles Embedded in a DNA Honeycomb Lattice

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Sequence Requirements for Plasmid Nuclear Import

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Structure–activity relationships of poly(l-lysines): effects of pegylation and molecular shape on physicochemical and biological properties in gene delivery

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Intravenously administered gold nanoparticles pass through the blood–retinal barrier depending on the particle size, and induce no retinal toxicity

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Molecular Genetics of Human Retinal Disease

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Mitosis enhances transgene expression of plasmid delivered by cationic liposomes

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Preparation and Evaluation of Thiol-Modified Gelatin Nanoparticles for Intracellular DNA Delivery in Response to Glutathione

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Intravitreal injection of AAV2 transduces macaque inner retina

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Gold nanoparticles delivery in mammalian live cells: a critical review

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Progress and prospects: nuclear import of nonviral vectors

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Efficient Non-Viral Ocular Gene Transfer with Compacted DNA Nanoparticles

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Antibiotic-free nanotherapeutics: Ultra-small, mucus-penetrating solid lipid nanoparticles enhance the pulmonary delivery and anti-virulence efficacy of novel quorum sensing inhibitors

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Curcumin nanoparticles attenuate neurochemical and neurobehavioral deficits in experimental model of huntington’s disease.

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Intravenous transferrin, RGD peptide and dual-targeted nanoparticles enhance anti-VEGF intraceptor gene delivery to laser-induced CNV

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A Trojan horse biomimetic delivery system using mesenchymal stem cells for HIF-1α siRNA-loaded nanoparticles on retinal pigment epithelial cells under hypoxia environment

Zhang, L., Wang, H., Li, M.-Q., Wang, X., Fan, L., & Wang, Y. (2022) · International Journal of Ophthalmology · doi:10.18240/ijo.2022.11.03 · Cited by 6 · see citing works

Toward a new frontier in diabetic retinopathy treatment: A synergistic approach using gene therapy and nanotechnology

Diress, M., Ionescu, C. M., Foster, T., Lim, P., Sen, L. Y., Kovačević, B., Wagle, S. R., Mooranian, A., & Al‐Salami, H. (2025) · Journal of Drug Delivery Science and Technology · doi:10.1016/j.jddst.2025.107172 · Cited by 5 · see citing works

Multimodal Imaging in Stem Cell Therapy for Retinal Disease

Zheng, M., & Paulus, Y. M. (2025) · Photonics · doi:10.3390/photonics12050413 · Cited by 4 · see citing works

Preparation of Targeted Mitochondrion Nanoscale-Release Peptides and Their Efficiency on Eukaryotic Cells

He, Y., Quan, Z., Zhang, R., He, B., Xu, Y. L., Chen, Z., Ren, Y., & Li, K. (2021) · Journal of Biomedical Nanotechnology · doi:10.1166/jbn.2021.3141 · Cited by 4 · see citing works

Application Prospects for Synthetic Nanoparticles in Optogenetic Retinal Prosthetics

Rotov, A. Y., Romanov, I. S., Tarakanchikova, Y. V., & Astakhova, L. A. (2021) · Journal of Evolutionary Biochemistry and Physiology · doi:10.1134/s0022093021060132 · Cited by 4 · see citing works

Retinal gene therapies for inherited ocular diseases: Translational delivery strategies from bench to bedside

Haddadzadegan, S., Mehdizdeh, N., Wang, X., Wong, T. T. L., & Miserez, A. (2026) · Journal of Controlled Release · doi:10.1016/j.jconrel.2026.114841 · Cited by 3 · see citing works

The next‐generation therapies in ophthalmology for blindness worldwide

Xu, J., Zhang, C., Jiang, J., & Jin, Z. (2024) · Eye & ENT Research · doi:10.1002/eer3.4 · Cited by 3 · see citing works

The Contribution of Nanomedicine in Ocular Oncology

Spyratou, E., Margarita, T., Droulias, A., Zachou, M.-E., & EFSTATHOPOULOS, E. P. (2025) · Cancers · doi:10.3390/cancers17071186 · Cited by 3 · see citing works

Gene Therapy for Retinal Diseases

Gopinath, C., Sarkar, S., & Ghosh, A. (2021) · Essentials in ophthalmology/Essentials ophthalmology · doi:10.1007/978-981-15-9184-6_30 · Cited by 3 · see citing works

Molecular Therapy for Choroideremia: Pre-clinical and Clinical Progress to Date

Kalatzis, V., Roux, A., & Meunier, I. (2021) · Molecular Diagnosis & Therapy · doi:10.1007/s40291-021-00558-y · Cited by 3 · see citing works

Nucleic Acids–based Bionanomaterials for Drug and Gene Therapy

Stoleru, E., & Vasile, C. (2019) · Elsevier eBooks · doi:10.1016/b978-0-12-813932-5.00006-6 · Cited by 3 · see citing works

Lipid nanoparticle mediated delivery of dsDNA to the murine retina

Fehrman, R. L., Sahel, D. K., Sahay, G., Lipinski, D. M., & Ryals, R. C. (2025) · Journal of Controlled Release · doi:10.1016/j.jconrel.2025.114194 · Cited by 2 · see citing works

Recent advances of novel nanoformulations combatting blue light hazards in the retina

Ghosh, S., Parua, P., Jana, K., Ghosh, S. K., Ghosh, A., Debnath, B., Halder, J., Sahoo, R. K., Rai, V. K., Dash, P., et al. (2025) · Experimental Eye Research · doi:10.1016/j.exer.2025.110806 · Cited by 2 · see citing works

mRNA delivery to the retina restores REP1 function in choroideremia

de Lemos, L., Antas, P., Carvalho, C., Castro, M., Bruno, D., Fonseca, A. F., Rakshit, S., & Seabra, M. C. (2026) · Molecular Therapy Advances · doi:10.1016/j.omta.2026.201768 · Cited by 2 · see citing works

Stem cells and genetic engineering empower therapeutic development for blinding eye diseases

Zhang, C.-J., Jin, K., & Jin, Z. (2023) · Elsevier eBooks · doi:10.1016/b978-0-323-99987-8.00009-6 · Cited by 2 · see citing works

Nanotechnology in retinal diseases: From disease diagnosis to therapeutic applications

Kaur, G., Bisen, S., & Singh, N. K. (2024) · Biophysics Reviews · doi:10.1063/5.0214899 · Cited by 2 · see citing works

Virus 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

Nanoparticles as drug delivery agents for managing diabetic retinopathy

Mukherjee, S., Panda, P., & Mishra, M. (2023) · Elsevier eBooks · doi:10.1016/b978-0-12-820557-0.00014-x · Cited by 2 · see citing works

Terapötik Moleküllerin Aktarımında Kullanılan Yeni Nesil Biyomalzemeler

PAMUKCI, A., Portakal, H. S., & Eroğlu, E. (2018) · Erzincan Üniversitesi Fen Bilimleri Enstitüsü Dergisi · doi:10.18185/erzifbed.339405 · Cited by 2 · see citing works

A Trojan horse biomimetic delivery system using mesenchymal stem cells for HIF-1α siRNA -loaded nanoparticles on RPE cells under hypoxia environmentPreprint

Zhang, L., Yan, J., Li, M.-Q., Wang, X., Li, F., Wang, H., & Wang, Y. (2022) · Research Square · doi:10.21203/rs.3.rs-1385244/v1 · Cited by 2 · see citing works

In vitro and in vivo toxicity of metal nanoparticles and their drug delivery applications

Jeevanandam, J., & Chan, Y. S. (2022) · Elsevier eBooks · doi:10.1016/b978-0-12-824408-1.00004-1 · Cited by 1 · see citing works

In vitro evaluation of a cationic polymer-lipid conjugate as a potential nanosystem for gene delivery

Sikhosana, N., du Toit, L. C., Fru, P., Walvekar, P., & Choonara, Y. E. (2026) · International Journal of Pharmaceutics · doi:10.1016/j.ijpharm.2026.126641 · Cited by 1 · see citing works

A Review of Personalised Molecular Medicine for the Treatment of Corneal Disorders

Courtney, D. G., Thakur, A., Ma, N., Moore, J. E., & Moore, T. (2015) · International Journal of Ophthalmology & Eye Science · doi:10.19070/2332-290x-si02002 · Cited by 1 · see citing works

Recent Developments in Maculopathy

Bandello, F. M., Battista, M., Brambati, M., Starace, V., Arrigo, A., & Parodi, M. B. (2019) · Springer eBooks · doi:10.1007/978-3-030-25389-9_6 · Cited by 1 · see citing works

Therapeutic Benefits from Nanoparticles: The Potential Significance of Nanoscience in Retinal Degenerative Diseases.

Rajala, R. V. S. (2019) · PubMed · Cited by 1 · see citing works

siRNA Mediated Gene Silencing in the Pancreatic Cancer Capan-1 Cell Line

Yıldıran, F. A. B. (2022) · Türk doğa ve fen dergisi :/Türk doğa ve fen dergisi · doi:10.46810/tdfd.1097432 · Cited by 1 · see citing works

RPE and Gene Therapy

Stieger, K., & Lorenz, B. (2020) · Springer eBooks · doi:10.1007/978-3-030-28384-1_15 · Cited by 2 · see citing works

Introduction to Organ Fabrication

Birla, R. K. (2016) · Learning materials in biosciences · doi:10.1007/978-3-319-41504-8_1

Corneal Haze, Refractive Surgery, and Implications for Choroidal Neovascularization

Singh, A., Abd, A. J., Al-Mashahedah, A. M. I., & Kanwar, J. R. (2018) · Springer eBooks · doi:10.1007/978-3-319-95807-1_25

Nanomedicine-Based Gene Delivery for the Retina and Posterior Segment Diseases

Lalwani, A., Shelat, P., & Patel, J. K. (2018) · Springer eBooks · doi:10.1007/978-3-319-95807-1_16

Molecular insights into PCARE-associated retinal disease

Serrano, J. C. C. (2018) · Radboud Repository (Radboud University)

Ocular Tissue Engineering

Karamichos, D. (2016) · MDPI eBooks · doi:10.3390/books978-3-03842-202-0

Nanoparticles in Ophthalmology

(2022) · Journal of Ophthalmology & Clinical Research · doi:10.33140/jocr.06.03.01

Optogenetic Prosthetization of Retinal Bipolar Cells

Rotov, A. Y., & Firsov, M. L. (2022) · Journal of Evolutionary Biochemistry and Physiology · doi:10.1134/s0022093022060011

Role of nanoparticles in pancreatic drug delivery: Including a bibliography on Targeted drug delivery

Sharma, N., Arora, V., Joshi, G., Prajapati, B. G., & Parihar, A. (2023) · IP International Journal of Comprehensive and Advanced Pharmacology · doi:10.18231/j.ijcaap.2023.019

RPE und Gentherapie

Stieger, K., & Lorenz, B. (2024) · Springer eBooks · doi:10.1007/978-3-031-35055-9_15

Applications of Nanofillers in the Biomedical Industry

Fahmy, H. M., Hegazy, A. S., Elbadry, A. M. M., Soliman, R. I., Bassiony, M., Mahmoud, S. M., & Ashry, H. (2024) · Springer eBooks · doi:10.1007/978-981-99-3516-1_55-1

Application of Nanomaterials in Nanomedicine

Islam, N., & Bag, A. (2025) · Smart nanomaterials technology · doi:10.1007/978-981-96-2112-5_3

In Vitro Gene Delivery in Retinal Pigment Epithelium Cells by Plasmid DNA-Wrapped Gold Nanoparticles

Trigueros, S., Domènech, E. B., Toulis, V., & Marfany, G. (2019) · Genes · doi:10.3390/genes10040289 · Cited by 45 · see citing works

Nanoparticles as Delivery Vehicles for the Treatment of Retinal Degenerative Diseases

Wang, Y., Rajala, A., & Rajala, R. V. S. (2018) · Advances in experimental medicine and biology · doi:10.1007/978-3-319-75402-4_15 · Cited by 11 · see citing works

Nanocarrier-Mediated Gene Delivery for Treatment of Inherited Retinal Disorders

Nguyen, M., Tran, T., & Le, L. (2025) · Zenodo (CERN European Organization for Nuclear Research) · doi:10.5281/zenodo.22849440

Formulation and Evaluation of Polymer‐Based Nanoparticles for Intravitreal Gene‐Delivery Applications

Lee, D., Kwak, G., Johnson, T. V., & Suk, J. S. (2022) · Current Protocols · doi:10.1002/cpz1.607 · Cited by 2 · see citing works

Mesoporous Silica-Based Nanoparticles as Non-Viral Gene Delivery Platform for Treating Retinitis Pigmentosa

Valdés-Sánchez, L., Borrego-González, S., Montero-Sánchez, A., Massalini, S., de la Cerda, B., Díaz‐Cuenca, A., & Diaz-Corrales, F. J. J. (2022) · Journal of Clinical Medicine · doi:10.3390/jcm11082170 · Cited by 26 · see citing works