CasRx-mediated RNA targeting prevents choroidal neovascularization in a mouse model of age-related macular degeneration
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Abstract
Abstract The smallest Cas13 family protein, CasRx, has a high cleavage activity and targeting specificity, offering attractive opportunity for therapeutic applications. Here we report that delivery of CasRx by adeno-associated virus via intravitreal injection could efficiently knockdown Vegfa transcripts and significantly reduce the area of laser-induced choroidal neovascularization in a mouse model of age-related macular degeneration. Thus, RNA-targeting CRISPR system could be used for in vivo gene therapy.
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Previous studies have focused on specific cell types believed to be major contributors to VEGFA expression, like RPE and Müller cells.
“previous studies have focused on specific cell types that are believed to be major contributors to VEGFA expression, like RPE and Müller cells (4, 43).”
From Characterization of RNA editing and gene therapy with a compact CRISPR-Cas13 in the retina (2024) · cites this paper as [4]
The paper’s Abstract describes Vegfa knockdown and analysis of a choroid–retinal pigment epithelial tissue complex, but does not say that previous studies focused on RPE or Müller cells as major contributors to VEGFA expression. Searches for those cell types and for cell-specific VEGFA targeting found no passage addressing that claim; no page number is provided in our copy. (LLM, conf=0.72)
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Cites
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Cited by
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High-fidelity Cas13 variants for targeted RNA degradation with minimal collateral effects
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Characterization of RNA editing and gene therapy with a compact CRISPR-Cas13 in the retina
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Optimization of specific RNA knockdown in mammalian cells with CRISPR-Cas13
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- Authors
- Zhou, C., Hu, X., Tang, C., Liu, W., Wang, S., Zhou, Y., Zhao, Q., Bo, Q., Shi, L., Sun, X., Zhou, H., & Yang, H.
- Affiliations
- Chinese Academy of Sciences (CN)
- Shanghai Jiao Tong University (CN)
- Corresponding author
- Sun, Xiaodong
- Zhou, Haibo
- Yang, Hui
- ORCID
- Venue
- National Science Review
- Publisher
- Oxford University Press (OUP)
- Published
- 2020-03-03
- Volume
- 7
- Issue
- 5
- Pages
- 835-837
- ISSN
- 2053-714X (ISSN-L)
- 2095-5138
- PMC
- PMC8288881
- OpenAlex
- W3010578254
- Cited by
- 69
- References
- 3
- Citations by year
- 2026: 9 · 2025: 9 · 2024: 14 · 2023: 13 · 2022: 13 · 2021: 7 · 2020: 4
- FWCI
- 3.49
- Type
- journal article
- Language
- en
- Open access
- gold
- doi.org
- Licence
- Locations
- National Science Review (publishedVersion, cc-by)
- bioRxiv (Cold Spring Harbor Laboratory) (acceptedVersion)
- PubMed Central (publishedVersion, cc-by)
- PubMed (publishedVersion)
- Funders
- R&D Program of China (2018YFC2000100, 2017YFC1001302)
- Strategic Priority Research Program (XDB32060000)
- National Natural Science Foundation of China (31871502, 31522037)
- Shanghai Municipal Science and Technology (2018SHZDZX05)
- Shanghai City Committee of science and technology (18411953700, 18JC1410100)
- Topics
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › CRISPR and Genetic Engineering
- Health Sciences › Medicine › Ophthalmology › Retinal Diseases and Treatments
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › RNA Interference and Gene Delivery
- Keywords
- Cas13, RNA-targeting CRISPR, choroidal neovascularization, age-related macular degeneration, laser-induced CNV, RNA interference, mouse model
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