ArticleMolecular therapy. Nucleic acids2017
In Vivo Knockout of the Vegfa Gene by Lentiviral Delivery of CRISPR/Cas9 in Mouse Retinal Pigment Epithelium Cells.
Article in Molecular therapy. Nucleic acids, 2017. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 43 papers.
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Who cites it
43 citing papers in PubMed, 73 citations in OpenAlex.
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- AAV-mediated multiple gene therapy combining VEGFA-targeting miR-agshRNAs and PEDF for the suppression of choroidal neovascularization.Molecular therapy. Nucleic acids · 2026Article
- Activation of the pituitary adenylate cyclase-activating polypeptide type I receptor promotes neuroblastoma proliferation and migration through distinct G protein pathways.Cell communication and signaling : CCS · 2026Article
- Harnessing the evolving CRISPR/Cas9 for precision oncology.Journal of translational medicine · 2024Review
- Multifunctional nano-in-micro delivery systems for targeted therapy in fundus neovascularization diseases.Journal of nanobiotechnology · 2024Review
- Benefits and Caveats in the Use of Retinal Pigment Epithelium-Specific Cre Mice.International journal of molecular sciences · 2024Review
- Pristimerin suppresses AIM2 inflammasome by modulating AIM2-PYCARD/ASC stability via selective autophagy to alleviate tendinopathy.Autophagy · 2024Article
- Therapeutic applications of CRISPR/Cas9 gene editing technology for the treatment of ocular diseases.The FEBS journal · 2023Review
- Engineered lentivirus-derived nanoparticles (LVNPs) for delivery of CRISPR/Cas ribonucleoprotein complexes supporting base editing, prime editing and in vivo gene modification.Nucleic acids research · 2023Article
- Genome editing in the treatment of ocular diseases.Experimental & molecular medicine · 2023Review
- CRISPR/Cas9 mediated specific ablation of vegfa in retinal pigment epithelium efficiently regresses choroidal neovascularization.Scientific reports · 2023Article
- Optimization of CRISPR-Cas system for clinical cancer therapy.Bioengineering & translational medicine · 2023Review
- Systemic Cytokines in Retinopathy of Prematurity.Journal of personalized medicine · 2023Review
- CRISPR/Cas systems usher in a new era of disease treatment and diagnosis.Molecular biomedicine · 2022Review
- Delivering genes with human immunodeficiency virus-derived vehicles: still state-of-the-art after 25 years.Journal of biomedical science · 2022Review
- New Aspects on the Treatment of Retinopathy of Prematurity: Currently Available Therapies and Emerging Novel Therapeutics.International journal of molecular sciences · 2022Review
- Lentiviral Vectors for Ocular Gene Therapy.Pharmaceutics · 2022Review
- CRISPR-based VEGF suppression using paired guide RNAs for treatment of choroidal neovascularization.Molecular therapy. Nucleic acids · 2022Article
- The inhibition of protein translation promotes tumor angiogenic switch.Molecular biomedicine · 2022Article
- Review
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Authors and funding
8 authors at 2 institutions in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Virus-based gene therapy by CRISPR/Cas9-mediated genome editing and knockout may provide a new option for treatment of inherited and acquired ocular diseases of the retina. In support of this notion, we show that Streptococcus pyogenes (Sp) Cas9, delivered by lentiviral vectors (LVs), can be used in vivo to selectively ablate the vascular endothelial growth factor A (Vegfa) gene in mice. By generating LVs encoding SpCas9 targeted to Vegfa, and in parallel the fluorescent eGFP marker protein, we demonstrate robust knockout of Vegfa that leads to a significant reduction of VEGFA protein in transduced cells. Three of the designed single-guide RNAs (sgRNAs) induce in vitro indel formation at high frequencies (44%-93%). A single unilateral subretinal injection facilitates RPE-specific localization of the vector and disruption of Vegfa in isolated eGFP
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