ArticleScience advances2018
Endothelial siRNA delivery in nonhuman primates using ionizable low-molecular weight polymeric nanoparticles.
Article in Science advances, 2018. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 58 papers.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
58 citing papers in PubMed, 115 citations in OpenAlex.
- Delivery Systems for Therapeutic Genome Editing: Challenges, Innovations, and Future Perspectives.MedComm · 2026Review
- How Advanced is Nanomedicine to Treat Atherosclerosis? A Comprehensive Review of the Literature.International journal of nanomedicine · 2026Review
- Lipid Nanoparticles for Delivery of CRISPR Gene Editing Components.Small methods · 2026Review
- Intravenous lipid-siRNA conjugate mediates gene silencing at the blood-brain barrier and blood-CSF barrier.Journal of controlled release : official journal of the Controlled Release Society · 2025Article
- Activation of the coagulation cascade as a mechanism for selective nanoparticle-mediated RNA delivery to the endothelium in vivo.Science advances · 2025Article
- Nanomedicine in Cancer Therapeutics: Current Perspectives from Bench to Bedside.Molecular cancer · 2025Review
- Nanomedicine-based strategies for the treatment of vein graft disease.Nature reviews. Cardiology · 2025Review
- Ligand-free biodegradable poly(beta-amino ester) nanoparticles for targeted systemic delivery of mRNA to the lungs.Biomaterials · 2025Article
- The future of siRNA-mediated approaches to treat von Willebrand disease.Expert review of hematology · 2025Review
- Research progress on the application of nanoparticles delivery in the treatment of atherosclerosis: implications for therapeutic interventions.Frontiers in cell and developmental biology · 2025Review
- Silencing of the von Willebrand factor gene in proatherothromboticResearch and practice in thrombosis and haemostasis · 2025Article
- mRNA cancer vaccines from bench to bedside: a new era in cancer immunotherapy.Biomarker research · 2024Review
- ROS-sensitive PD-L1 siRNA cationic selenide nanogels for self-inhibition of autophagy and prevention of immune escape.Bioactive materials · 2024Article
- mRNA vaccines in tumor targeted therapy: mechanism, clinical application, and development trends.Biomarker research · 2024Review
- Effective delivery of miR-150-5p with nucleus pulposus cell-specific nanoparticles attenuates intervertebral disc degeneration.Journal of nanobiotechnology · 2024Article
- Influencing Endothelial Cells' Roles in Inflammation and Wound Healing Through Nucleic Acid Delivery.Tissue engineering. Part A · 2024Review
- Recent trends in the delivery of RNA drugs: Beyond the liver, more than vaccine.European journal of pharmaceutics and biopharmaceutics : official journal of Arbeitsgemeinschaft fur Pharmazeutische Verfahrenstechnik e.V · 2024Review
- An immunosuppressive vascular niche drives macrophage polarization and immunotherapy resistance in glioblastoma.Science advances · 2024Article
- Protein interaction networks in the vasculature prioritize genes and pathways underlying coronary artery disease.Communications biology · 2024Article
- Cationic LNP-formulated mRNA expressing Tie2-agonist in the lung endothelium prevents pulmonary vascular leakage.Molecular therapy. Nucleic acids · 2023Article
Corrections and comments
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Authors and funding
10 authors at 2 institutions in 1 country.
Funding
Abstract
Dysfunctional endothelial cells contribute to the pathophysiology of many diseases, including vascular disease, stroke, hypertension, atherosclerosis, organ failure, diabetes, retinopathy, and cancer. Toward the goal of creating a new RNA-based therapy to correct aberrant endothelial cell gene expression in humans, efficient gene silencing in the endothelium of nonhuman primates was achieved by delivering small interfering RNA (siRNA) with 7C1, a low-molecular weight, ionizable polymer that forms nanoparticles. After a single intravenous administration of 1 mg of siRNA per kilogram of animal, 7C1 nanoparticles delivering Tie2 siRNA caused Tie2 mRNA levels to decrease by approximately 80% in the endothelium of the lung. Significant decreases in Tie2 mRNA were also found in the heart, retina, kidney, pancreas, and bone. Blood chemistry and liver function analysis before and after treatment all showed protein and enzyme concentrations within the normal reference ranges. Furthermore, after controlling for siRNA-specific effects, no significant increases in inflammatory cytokine concentrations were found in the serum. Similarly, no gross lesions or significant underlying pathologies were observed after histological examination of nonhuman primate tissues. This study is the first demonstration of endothelial gene silencing in multiple nonhuman primate organs using systemically administered siRNA nanoparticles and highlights the potential of this approach for the treatment of disease in humans.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.