ArticleMolecular therapy : the journal of the American Society of Gene Therapy2010
Noninvasive imaging of lipid nanoparticle-mediated systemic delivery of small-interfering RNA to the liver.
Article in Molecular therapy : the journal of the American Society of Gene Therapy, 2010. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 23 papers.
What it found
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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.
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Who cites it
23 citing papers in PubMed, 56 citations in OpenAlex.
- RNAi-based drug design: considerations and future directions.Nature reviews. Drug discovery · 2024Review
- Regulatory guidelines and preclinical tools to study the biodistribution of RNA therapeutics.Advanced drug delivery reviews · 2022Review
- Nanoparticle Delivery Platforms for RNAi Therapeutics Targeting COVID-19 Disease in the Respiratory Tract.International journal of molecular sciences · 2022Review
- Lipid nanoparticles for delivery of RNA therapeutics: Current status and the role ofTheranostics · 2022Review
- Lipids and Lipid Derivatives for RNA Delivery.Chemical reviews · 2021Review
- Article
- A Direct Comparison of in Vitro and in Vivo Nucleic Acid Delivery Mediated by Hundreds of Nanoparticles Reveals a Weak Correlation.Nano letters · 2018Article
- Delivery of therapeutic siRNA to the lung endothelium via novel Lipoplex formulation DACC.Molecular therapy : the journal of the American Society of Gene Therapy · 2014Article
- Nanoparticle delivery of antisense oligonucleotides and their application in the exon skipping strategy for Duchenne muscular dystrophy.Nucleic acid therapeutics · 2014Review
- Technologies for investigating the physiological barriers to efficient lipid nanoparticle-siRNA delivery.The journal of histochemistry and cytochemistry : official journal of the Histochemistry Society · 2013Review
- Multimodality imaging of RNA interference.Current medicinal chemistry · 2013Review
- Human serum albumin-coated lipid nanoparticles for delivery of siRNA to breast cancer.Nanomedicine : nanotechnology, biology, and medicine · 2013Article
- Cystamine-terminated poly(beta-amino ester)s for siRNA delivery to human mesenchymal stem cells and enhancement of osteogenic differentiation.Biomaterials · 2012Article
- Lipid-based vectors for siRNA delivery.Journal of drug targeting · 2012Review
- Targeted siRNA Delivery and mRNA Knockdown Mediated by Bispecific Digoxigenin-binding Antibodies.Molecular therapy. Nucleic acids · 2012Article
- Cationic lipid-coated gold nanoparticles as efficient and non-cytotoxic intracellular siRNA delivery vehicles.Pharmaceutical research · 2012Article
- Delivery of siRNA to the mouse lung via a functionalized lipopolyamine.Molecular therapy : the journal of the American Society of Gene Therapy · 2012Article
- Persistent dystrophin protein restoration 90 days after a course of intraperitoneally administered naked 2'OMePS AON and ZM2 NP-AON complexes in mdx mice.Journal of biomedicine & biotechnology · 2012Article
- Bioluminescence imaging: progress and applications.Trends in biotechnology · 2011Review
- Biodistribution of small interfering RNA at the organ and cellular levels after lipid nanoparticle-mediated delivery.The journal of histochemistry and cytochemistry : official journal of the Histochemistry Society · 2011Article
Corrections and comments
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Authors and funding
9 authors at 1 institution in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Mouse models with liver-specific expression of firefly luciferase were developed that enable a noninvasive and longitudinal assessment of small-interfering RNA (siRNA)-mediated gene silencing in hepatocytes of live animals via bioluminescence imaging. Using these models, a set of lipid nanoparticles (LNPs) with different compositions of cationic lipids, polyethylene glycol (PEG), and cholesterol, were tested for their abilities in delivering a luciferase siRNA to the liver via systemic administration. A dose-dependent luciferase knockdown by LNP/siRNA assemblies was measured by in vivo bioluminescence imaging, which correlated well with the results from parallel ex vivo analyses of luciferase mRNA and protein levels in the liver. RNA interference (RNAi)-mediated target silencing was further confirmed by the detection of RNAi-specific target mRNA cleavage. A single dose of LNP02L at 3 mg/kg (siRNA) caused 90% reduction of luciferase expression and the target repression lasted for at least 10 days. With identical components, LNPs containing 2% PEG are more potent than those with 5.4% PEG. Our results demonstrate that these liver-luciferase mouse models provide a powerful tool for a high-throughput evaluation of hepatic delivery platforms by noninvasive imaging and that the molar ratio of PEG lipid can affect the efficacy of LNPs in silencing liver targets via systemic administration.
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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.