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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.
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“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]
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“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]
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InfluentialGene Therapy for Retinal Diseases
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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
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