A Partial Structural and Functional Rescue of a Retinitis Pigmentosa Model with Compacted DNA Nanoparticles
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Previously we have shown that compacted DNA nanoparticles can drive high levels of transgene expression after subretinal injection in the mouse eye. Here we delivered compacted DNA nanoparticles containing a therapeutic gene to the retinas of a mouse model of retinitis pigmentosa. Nanoparticles containing the wild-type retinal degeneration slow (Rds) gene were injected into the subretinal space of rds(+/-) mice on postnatal day 5. Gene expression was sustained for up to four months at levels up to four times higher than in controls injected with saline or naked DNA. The nanoparticles were taken up into virtually all photoreceptors and mediated significant structural and biochemical rescue of the disease without histological or functional evidence of toxicity. Electroretinogram recordings showed that nanoparticle-mediated gene transfer restored cone function to a near-normal level in contrast to transfer of naked plasmid DNA. Rod function was also improved. These findings demonstrate that compacted DNA nanoparticles represent a viable option for development of gene-based interventions for ocular diseases and obviate major barriers commonly encountered with non-viral based therapies.
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Single-molecule DNA nanoparticles compacted by CK30-PEG have been used to carry payloads up to 20 kb in size, with several groups demonstrating their safety in mouse models of retinal degeneration.
“Single-molecule DNA nanoparticles compacted by polyethylene glycol (PEG)-substituted lysine peptides (CK30-PEG) have been used to carry payloads up to 20 kb in size,82,83 with several groups demonstrating their safety in BALB/c mice and rds+/− Rho-/- or Abca4-/- mouse models of retinal degeneration.82–86”
From Gene Therapy for Retinal Degenerative Diseases: Progress, Challenges, and Future Directions (2023) · cites this paper as [82]
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- Authors
- Cai, X., Nash, Z., Conley, S. M., Fliesler, S. J., Cooper, M. J., & Naash, M. I.
- Affiliations
- University of Oklahoma Health Sciences Center (US)
- Saint Louis University (US)
- Copernicus Therapeutics (United States) (US)
- Corresponding author
- Naash, Muna I.
- Venue
- PLoS ONE
- Publisher
- Public Library of Science (PLoS)
- Published
- 2009-04-24
- Volume
- 4
- Issue
- 4
- Pages
- e5290
- ISSN
- 1932-6203 (ISSN-L)
- PMC
- PMC2669177
- OpenAlex
- W1969461625
- Cited by
- 109
- References
- 49
- Citations by year
- 2026: 2 · 2025: 8 · 2024: 3 · 2023: 3 · 2022: 5 · 2021: 5 · 2020: 5 · 2019: 1 · 2018: 7 · 2017: 4 · 2016: 4 · 2015: 7 · 2014: 8 · 2013: 8 · 2012: 14
- FWCI
- 5.24
- Type
- journal article
- Language
- en
- Open access
- gold
- doi.org
- Locations
- PLoS ONE (publishedVersion, cc-by)
- PubMed Central (publishedVersion, cc-by)
- PubMed (publishedVersion)
- DOAJ (DOAJ: Directory of Open Access Journals) (submittedVersion, cc-by-sa)
- Figshare (submittedVersion, cc-by)
- Funders
- Foundation Fighting Blindness
- Research to Prevent Blindness
- Oklahoma Center for the Advancement of Science and Technology
- National Institutes of Health (EY018656)
- National Eye Institute (R56EY010609, EY007361, EY10609, R01EY007361, R01EY018656, R01EY010609, EY018656)
- Topics
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › Retinal Development and Disorders
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › RNA Interference and Gene Delivery
- Life Sciences › Pharmacology, Toxicology and Pharmaceutics › Pharmaceutical Science › Advanced Drug Delivery Systems
- Keywords
- retinitis pigmentosa, gene therapy, subretinal injection, photoreceptors, cone function, rod function, electroretinogram, non-viral gene delivery, ocular gene therapy, mouse model
- MeSH terms
- Animals, Disease Models, Animal, DNA, Electroretinography, Immunohistochemistry, Intermediate Filament Proteins, Membrane Glycoproteins, Mice, Transgenic, Nerve Tissue Proteins, Retinitis Pigmentosa, Transgenes, Photoreceptor Cells, Vertebrate, Mice, Nanoparticles, Peripherins
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