The DOI matches its Crossref record; 236 of the 240 references it cites were found in a registry or an index.
Nanoparticle-based technologies for retinal gene therapy
Open accessAbstract
For patients with hereditary retinal diseases, retinal gene therapy offers significant promise for the prevention of retinal degeneration. While adeno-associated virus (AAV)-based systems remain the most popular gene delivery method due to their high efficiency and successful clinical results, other delivery systems, such as non-viral nanoparticles (NPs) are being developed as additional therapeutic options. NP technologies come in several categories (e.g., polymer, liposomes, peptide compacted DNA), several of which have been tested in mouse models of retinal disease. Here, we discuss the key biochemical features of the different NPs that influence how they are internalized into cells, escape from endosomes, and are delivered into the nucleus. We review the primary mechanism of NP uptake by retinal cells and highlight various NPs that have been successfully used for in vivo gene delivery to the retina and RPE. Finally, we consider the various strategies that can be implemented in the plasmid DNA to generate persistent, high levels of gene expression.
Overview
The claims and statistics of this paper haven’t been checked yet.
Run the check — free, no account needed. It reads the open-access text and checks the sources it cites.
Integrity signals
Registry
Cites
Statistics
Verification
Claims the paper makes
The claims and statistics of this paper haven’t been checked yet.
Run the check — free, no account needed. It reads the open-access text and checks the sources it cites.
Claims other papers make about this one
Each is checked against this paper’s own text: does it say what the citing paper says it does?
In the citing paper
“non-viral vectors have been explored for subretinal administration, they have generally been associated with toxicity and lower transduction levels compared to viral vectors [301].”
From Bruch’s Membrane: A Key Consideration with Complement-Based Therapies for Age-Related Macular Degeneration (2023) · cites this paper as [301]
In the citing paper
“Generally, these methods carry low risk of immune responses commonly associated with viral delivery [44,63,64].”
From Viral Delivery Systems for CRISPR (2019) · cites this paper as [64]
Self-check
Percentages, sums and reported differences re-computed from the paper’s own numbers.
The self-check needs the paper’s full text.
Citations
Cites
Items 201–236 of 236
Hollyfield, J. G., Varner, H. H., Rayborn, M. E., Liou, G. I., & Bridges, C. D. (1985) · The Journal of cell biology · doi:10.1083/jcb.100.5.1676 · Cited by 45
Contreras‐Ruiz, L., Zorzi, G. K., Hileeto, D., López‐García, A., Calonge, M., Seijo, B., Sánchez, A., & Diebold, Y. (2012) · Gene Therapy · doi:10.1038/gt.2012.56 · Cited by 45
Subretinal delivery of immunoglobulin G with gold nanoparticles in the rabbit eye
Hayashi, A., Naseri, A., Pennesi, M. E., & de Juan, E. (2009) · Japanese Journal of Ophthalmology · doi:10.1007/s10384-009-0655-x · Cited by 43
Delgado, D., del Pozo‐Rodríguez, A., Solinís, M. Á., Bartkowiak, A., & Rodríguez‐Gascón, A. (2013) · European Journal of Pharmaceutical Sciences · doi:10.1016/j.ejps.2013.08.013 · Cited by 42
Molecular and Biochemical Characterization of Folate Transport Proteins in Retinal Müller Cells
Bozard, B. R., Ganapathy, P. S., Duplantier, J., Mysona, B. A., Ha, Y., Roon, P., Smith, R. M., Goldman, I. D., Prasad, P. D., Martin, P. M., et al. (2010) · Investigative Ophthalmology & Visual Science · doi:10.1167/iovs.09-4833 · Cited by 42
Vogel, V., Lochmann, D., Weyermann, J., Mayer, G., Tziatzios, C., van den Broek, J. A., Haase, W., Wouters, D., Schubert, U. S., Kreuter, J., et al. (2005) · Journal of Controlled Release · doi:10.1016/j.jconrel.2004.11.029 · Cited by 42
Han, Z., Koirala, A., Makkia, R. S., Cooper, M. J., & Naash, M. I. (2012) · Nanomedicine · doi:10.2217/nnm.11.158 · Cited by 40
Self-citationHan, Z., Conley, S. M., & Naash, M. I. (2011) · Investigative Ophthalmology & Visual Science · doi:10.1167/iovs.10-6916 · Cited by 40
Self-citationArima, H., Arizono, M., Higashi, T., Yoshimatsu, A., Ikeda, H., Motoyama, K., HATTORI, K., Takeuchi, T., Hirayama, F., & Uekama, K. (2012) · Cancer Gene Therapy · doi:10.1038/cgt.2012.9 · Cited by 36
Characteristics of DEAE-dextran-MMA graft copolymer as a nonviral gene carrier
Ōnishi, Y., Eshita, Y., Murashita, A., Mizuno, M., & Yoshida, J. (2007) · Nanomedicine Nanotechnology Biology and Medicine · doi:10.1016/j.nano.2007.07.002 · Cited by 36
Adeno-Associated Virus Mediated Gene Therapy for Retinal Degenerative Diseases
Stieger, K., Cronin, T., Bennett, J., & Rolling, F. (2011) · Methods in molecular biology · doi:10.1007/978-1-61779-370-7_8 · Cited by 35
Genini, S., Beltran, W. A., & Aguirre, G. D. (2013) · PLoS ONE · doi:10.1371/journal.pone.0085408 · Cited by 35
Yuan, H., He, S., Li, L., Yan, Zhang, Du, Y., Yu, & Hu, F.-Q. (2013) · International Journal of Nanomedicine · doi:10.2147/ijn.s47967 · Cited by 34
Oligonucleotide-protamine-albumin nanoparticles: Protamine sulfate causes drastic size reduction
Mayer, G., Vogel, V., Weyermann, J., Lochmann, D., van den Broek, J. A., Tziatzios, C., Haase, W., Wouters, D., Schubert, U. S., Zimmer, A., et al. (2005) · Journal of Controlled Release · doi:10.1016/j.jconrel.2005.04.019 · Cited by 34
Genomic cis-acting Sequences Improve Expression and Establishment of a Nonviral Vector
Hagedorn, C., Antoniou, M. N., & Lipps, H. J. (2013) · Molecular Therapy — Nucleic Acids · doi:10.1038/mtna.2013.47 · Cited by 33
Xu, J., & Amiji, M. M. (2012) · Journal of Visualized Experiments · doi:10.3791/3612 · Cited by 32
Jenke, A., Eisenberger, T., Baiker, A. E., Stehle, I. M., Wirth, S., & Lipps, H. J. (2005) · Human Gene Therapy · doi:10.1089/hum.2005.16.533 · Cited by 32
de Garibay, A. P. R., Solinís, M. Á., Rodríguez‐Gascón, A., Ganjian, H., & Fuchs, R. (2013) · The Journal of Gene Medicine · doi:10.1002/jgm.2749 · Cited by 28
Fei, Y., & Hughes, T. E. (2000) · PubMed · Cited by 27
Zhang, X., Yin, Y., Liu, -, M., Wang, & Hao (2012) · International Journal of Nanomedicine · doi:10.2147/ijn.s30671 · Cited by 27
Transportin Mediates Nuclear Entry of DNA in Vertebrate Systems
Lachish‐Zalait, A., Lau, C. K., Fichtman, B., Zimmerman, E., Harel, A., Gaylord, M. R., Forbes, D. J., & Elbaum, M. (2009) · Traffic · doi:10.1111/j.1600-0854.2009.00968.x · Cited by 27
Stenler, S., Andersson, A., Simonson, O. E., Lundin, K. E., Chen, Z.-Y., Kay, M. A., Smith, C. I. E., Sylvén, C., & Blomberg, P. (2009) · Journal of Cardiovascular Pharmacology · doi:10.1097/fjc.0b013e318194234e · Cited by 27
Vighi, E., Montanari, M., Ruozi, B., Tosi, G., Magli, A., & Leo, E. (2010) · European Journal of Pharmaceutics and Biopharmaceutics · doi:10.1016/j.ejpb.2010.07.012 · Cited by 27
Hollyfield, J. G., & Rayborn, M. E. (1987) · Experimental Eye Research · doi:10.1016/s0014-4835(87)80119-7 · Cited by 25
A designed curved DNA segment that is a remarkable activator of eukaryotic transcription
Sumida, N., Nishikawa, J., Kishi, H., Amano, M., Furuya, T., Sonobe, H., & Ohyama, T. (2006) · FEBS Journal · doi:10.1111/j.1742-4658.2006.05557.x · Cited by 25
Importin‐7 Mediates Nuclear Trafficking of DNA in Mammalian Cells
Dhanoya, A., Wang, T., Keshavarz‐Moore, E., Fassati, A., & Chain, B. (2012) · Traffic · doi:10.1111/tra.12021 · Cited by 22
Episomal Maintenance of S/MAR-Containing Non-Viral Vectors for RPE-Based Diseases
Koirala, A., Conley, S. M., & Naash, M. I. (2014) · Advances in experimental medicine and biology · doi:10.1007/978-1-4614-3209-8_88 · Cited by 19
Self-citationCell cycle dependent histone dynamics of an episomal non-viral vector
Rupprecht, S., & Lipps, H. J. (2009) · Gene · doi:10.1016/j.gene.2009.03.010 · Cited by 19
Mannose-sensitive HRP endocytosis by the retinal pigment epithelium
Heth, C. A., & Bernstein, M. H. (1991) · Experimental Eye Research · doi:10.1016/0014-4835(91)90130-7 · Cited by 18
Fukunaga, S., Kanda, G. N., Tanase, J.-I., Harashima, H., Ohyama, T., & Kamiya, H. (2011) · Gene Therapy · doi:10.1038/gt.2011.127 · Cited by 14
Transferrin- and folate-modified, double-targeted nanocarriers for gene delivery
Jing, F., Li, D., Xu, W., Liu, Y., Wang, K., & Sui, Z. (2013) · Pharmaceutical Biology · doi:10.3109/13880209.2013.853812 · Cited by 13
Rudolph, C., & Rosenecker, J. (2012) · Cold Spring Harbor Protocols · doi:10.1101/pdb.prot068122 · Cited by 12
Vighi, E., Leo, E., Montanari, M., Mucci, A., Hanuskovà, M., & Iannuccelli, V. (2011) · Therapeutic Delivery · doi:10.4155/tde.11.118 · Cited by 11
Delivery of 9-Cis Retinal to Photoreceptors from Bovine Serum Albumin
Li, Z., Zhuang, J., & Corson, D. W. (1999) · Photochemistry and Photobiology · doi:10.1111/j.1751-1097.1999.tb03319.x · Cited by 10
Three-Dimensional Molecular Imaging with Photothermal Optical Coherence Tomography
Skala, M. C., Crow, M. J., Wax, A. P., & Izatt, J. A. (2013) · Methods in molecular biology · doi:10.1007/978-1-62703-468-5_7 · Cited by 9
Cross, D. S., & Burmester, J. K. (2007) · Medical Oncology · doi:10.1007/s12032-007-0038-4 · Cited by 8
Flagged first, then by citations
4 of the 240 references couldn’t be matched to a known paper.
Research works printed but not linked: 1
- Hawley-Nelson, 1993, Lipofectamine reagent: a new, higher efficiency polycationic liposome transfection reagent, Focus, 73
Cited by
Sorted by citations
Progress in corneal wound healing
Ljubimov, A. V., & Saghizadeh, M. (2015) · Progress in Retinal and Eye Research · doi:10.1016/j.preteyeres.2015.07.002 · Cited by 900
“They are different in size, charge, and structure but are all able to enter the cells, while avoiding the endosomes, and transfer the vector into the nucleus for gene expression (Adijanto and Naash, 2015).”
BackgroundViral Delivery Systems for CRISPR
Xu, C. L., Ruan, M. Z. C., Mahajan, V. B., & Tsang, S. H. (2019) · Viruses · doi:10.3390/v11010028 · Cited by 277
“Generally, these methods carry low risk of immune responses commonly associated with viral delivery [44,63,64].”
BackgroundLipid nanoparticles for delivery of messenger RNA to the back of the eye
Patel, S., Ryals, R. C., Weller, K. K., Pennesi, M. E., & Sahay, G. (2019) · Journal of Controlled Release · doi:10.1016/j.jconrel.2019.04.015 · Cited by 260
Ocular Drug Delivery: Role of Degradable Polymeric Nanocarriers for Ophthalmic Application
Tsai, C.-H., Wang, P., Lin, I., Hu, H., Liu, G., & Tseng, C. (2018) · International Journal of Molecular Sciences · doi:10.3390/ijms19092830 · Cited by 241
“In general, NPs less than 250 nm are usually taken up by endocytosis [48,49].”
BackgroundInfluentialEarly and late stage gene therapy interventions for inherited retinal degenerations
Botto, C., Rucli, M., Tekinsoy, M. D., Pulman, J., Sahel, J.-A., & Dalkara, D. (2022) · Progress in Retinal and Eye Research · doi:10.1016/j.preteyeres.2021.100975 · Cited by 190
Metadata
- Authors
- Adijanto, J., & Naash, M. I.
- Affiliations
- University of Oklahoma Health Sciences Center (US)
- Corresponding author
- Naash, Muna I.
- ORCID
- Naash, Muna I.
- Venue
- European Journal of Pharmaceutics and Biopharmaceutics
- Publisher
- Elsevier BV
- Published
- 2015-09
- Volume
- 95
- Issue
- Pt B
- Pages
- 353-367
- ISSN
- 0939-6411 (ISSN-L)
- PMC
- PMC4499495
- OpenAlex
- W2045435902
- Cited by
- 107
- References
- 240
- Citations by year
- 2026: 4 · 2025: 12 · 2024: 9 · 2023: 6 · 2022: 12 · 2021: 14 · 2020: 6 · 2019: 13 · 2018: 8 · 2017: 11 · 2016: 4 · 2015: 8
- FWCI
- 1.00
- Type
- journal article
- Language
- en
- Open access
- green
- www.ncbi.nlm.nih.gov
- Locations
- European Journal of Pharmaceutics and Biopharmaceutics (publishedVersion)
- PubMed (publishedVersion)
- PubMed Central (acceptedVersion)
- Funders
- National Institutes of Health (EY18656, EY022778)
- Topics
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › RNA Interference and Gene Delivery
- Health Sciences › Medicine › Ophthalmology › Retinal Development and Disorders
- Life Sciences › Pharmacology, Toxicology and Pharmaceutics › Pharmaceutical Science › Advanced Drug Delivery Systems
- Keywords
- retinal gene therapy, nanoparticle-mediated gene delivery, inherited retinal disease, retinal degeneration, adeno-associated virus vectors, liposomes, nanoparticle uptake, endosomal escape, nuclear delivery, retinal pigment epithelium, persistent gene expression, plasmid DNA
- MeSH terms
- Animals, Disease Models, Animal, DNA, Gene Expression Regulation, Humans, Retina, Retinal Diseases, Genetic Therapy, Gene Transfer Techniques, Nanotechnology, Mice, Nanoparticles
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 ›
Are you an author, or do you see a problem with this page? Request a review
