ArticleMolecular therapy : the journal of the American Society of Gene Therapy2014
Delivery of therapeutic siRNA to the lung endothelium via novel Lipoplex formulation DACC.
Article in Molecular therapy : the journal of the American Society of Gene Therapy, 2014. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 47 papers.
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Who cites it
47 citing papers in PubMed, 98 citations in OpenAlex.
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- Reaching Outer Space and Enabling the mRNA Revolution: A Critical Role of Partnerships for Successful Drug and Vaccine Development.Handbook of experimental pharmacology · 2024Review
- Delivery of nucleic acids using nanomaterials.Molecular biomedicine · 2023Review
- Cationic LNP-formulated mRNA expressing Tie2-agonist in the lung endothelium prevents pulmonary vascular leakage.Molecular therapy. Nucleic acids · 2023Article
- Delivery of gene editing therapeutics.Nanomedicine : nanotechnology, biology, and medicine · 2023Review
- Technological advances in the use of viral and non-viral vectors for delivering genetic and non-genetic cargos for cancer therapy.Drug delivery and translational research · 2023Review
- 'Passive' nanoparticles for organ-selective systemic delivery: design, mechanism and perspective.Chemical Society reviews · 2023Review
- Effect of the combination of cationic lipid and phospholipid on gene-knockdown using siRNA lipoplexes in breast tumor cells and mouse lungs.Molecular medicine reports · 2023Article
- Remodeling the tumor immune microenvironment via siRNA therapy for precision cancer treatment.Asian journal of pharmaceutical sciences · 2023Review
- Modified ASO conjugates encapsulated with cytidinyl/cationic lipids exhibit more potent and longer-lasting anti-HCC effects.Molecular therapy. Nucleic acids · 2023Article
- Lipid Nanoparticles for Nucleic Acid Delivery to Endothelial Cells.Pharmaceutical research · 2023Review
- Nanotechnology - a robust tool for fighting the challenges of drug resistance in non-small cell lung cancer.Beilstein journal of nanotechnology · 2023Review
- Nucleic Acid Delivery to the Vascular Endothelium.Molecular pharmaceutics · 2022Review
- Optimal combination of cationic lipid and phospholipid in cationic liposomes for gene knockdown in breast cancer cells and mouse lung using siRNA lipoplexes.Molecular medicine reports · 2022Article
- Lipid Nanoparticles for Organ-Specific mRNA Therapeutic Delivery.Pharmaceutics · 2021Review
- BRAF Modulates Stretch-Induced Intercellular Gap Formation through Localized Actin Reorganization.International journal of molecular sciences · 2021Article
- Effects of sterol derivatives in cationic liposomes on biodistribution and gene-knockdown in the lungs of mice systemically injected with siRNA lipoplexes.Molecular medicine reports · 2021Article
- miR-150-PTPMT1-cardiolipin signaling in pulmonary arterial hypertension.Molecular therapy. Nucleic acids · 2021Article
Corrections and comments
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Authors and funding
8 authors at 1 institution in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Posttranscriptional gene silencing by RNA interference can be therapeutically exploited to inhibit pathophysiological gene expression. However, in contrast to the established effectiveness of RNAi in vitro, safe and effective delivery of siRNAs to specific organs and cell types in vivo remains the major hurdle. Here, we report the development and in vivo characterization of a novel siRNA delivery system (DACC lipoplex) suitable for modulating target gene expression specifically in the lung vasculature. Systemic administration of DACC in mice delivered siRNA cargo functionally to the lung pulmonary endothelium. A single dose of DACC lipoplexes administered by bolus injection or by infusion was sufficient to specifically silence genes expressed in pulmonary endothelial cells such as CD31, Tie-2, VE-cadherin, or BMP-R2. When tested in a mouse model for lung cancer, repeated treatment with DACC/siRNA(CD31) reduced formation of lung metastases and increased life span in a mouse model of experimental lung metastasis.
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