Cited by 107RNA Interference and Gene DeliveryRetinal Development and DisordersAdvanced Drug Delivery Systems

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Nanoparticle-based technologies for retinal gene therapy

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Adijanto, Jeffrey, Naash, Muna I.·2015·European Journal of Pharmaceutics and Biopharmaceutics

DOI 10.1016/j.ejpb.2014.12.028PMC PMC4499495

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Abstract

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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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)
DOI: 10.1016/j.ejpb.2014.12.028
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
Licence: www.elsevier.com/tdm/userlicense/1.0/ (tdm)
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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