ArticleDrug delivery and translational research2014
Lipid nanoparticle delivery systems for siRNA-based therapeutics.
Article in Drug delivery and translational research, 2014. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 72 papers.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
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
72 citing papers in PubMed, 189 citations in OpenAlex.
- Structural evolution of ionizable lipids for nucleic acid delivery.Nature reviews. Chemistry · 2026Review
- Design and development of lipid nanoparticle formulations for brain gene therapy.Molecular therapy. Nucleic acids · 2026Article
- Ionizable Cholesterol-Integrated Lipid Nanoparticles for Efficient siRNA Delivery to Solid Tumors.ChemMedChem · 2026Article
- Review
- Functional-genomic prioritization identifiesFrontiers in pharmacology · 2026Article
- Dually Decorated Palmitate-Containing Lipid Nanoparticles for the Targeted Delivery of siRNAs against HER2 and Hsp27 in HER2Journal of medicinal chemistry · 2025Article
- Microrobotic Swarms for Cancer Therapy.Research (Washington, D.C.) · 2025Review
- Patent landscape of nanotechnology - based nucleic acid delivery systems for breast cancer therapies.Frontiers in oncology · 2025Article
- Overcoming antifungal resistance inFrontiers in cellular and infection microbiology · 2025Review
- siRNA-mediated therapeutic approaches improve acute kidney injury and limit its worsening.Frontiers in pharmacology · 2025Review
- Lipid Nanovesicles in Cancer Treatment: Improving Targeting and Stability of Antisense Oligonucleotides.Drug design, development and therapy · 2025Review
- Telomere Length and Telomerase Activity as Potential Biomarkers for Gastrointestinal Cancer.Cancers · 2024Review
- Development of an advanced separation and characterization platform for mRNA and lipid nanoparticles using multi-detector asymmetrical flow field-flow fractionation.Analytical and bioanalytical chemistry · 2024Article
- Nucleic Acid Therapeutics: Successes, Milestones, and Upcoming Innovation.Nucleic acid therapeutics · 2024Review
- Dyslipidemia: A Narrative Review on Pharmacotherapy.Pharmaceuticals (Basel, Switzerland) · 2024Review
- The landscape of nanoparticle-based siRNA delivery and therapeutic development.Molecular therapy : the journal of the American Society of Gene Therapy · 2024Review
- Lipid nanovehicles overcome barriers to systemic RNA delivery: Lipid components, fabrication methods, and rational design.Acta pharmaceutica Sinica. B · 2024Review
- Development of Lipid Nanoparticle Formulation for the Repeated Administration of mRNA Therapeutics.Biomaterials research · 2024Article
- pH-dependent structural transitions in cationic ionizable lipid mesophases are critical for lipid nanoparticle function.Proceedings of the National Academy of Sciences of the United States of America · 2023Article
- Immunogenicity of lipid nanoparticles and its impact on the efficacy of mRNA vaccines and therapeutics.Experimental & molecular medicine · 2023Review
12 more citing papers are in PubMed but not listed here.
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
3 authors at 2 institutions in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Therapeutics based on small interfering RNA (siRNA) have a huge potential for the treatment of disease but requires sophisticated delivery systems for in vivo applications. Lipid nanoparticles (LNP) are proven delivery systems for conventional small molecule drugs with over eight approved LNP drugs. Experience gained in the clinical development of LNP for the delivery of small molecules, combined with an understanding of the physical properties of lipids, can be applied to design LNP systems for in vivo delivery of siRNA. In particular, cationic lipids are required to achieve efficient encapsulation of oligonucleotides; however, the presence of a charge on LNP systems can result in toxic side effects and rapid clearance from the circulation. To address these problems, we have developed ionizable cationic lipids with pKa values below 7 that allow oligonucleotide encapsulation at low pH (e.g., pH 4) and a relatively neutral surface at physiological pH. Further optimization of cationic lipids to achieve maximized endosomal destabilization following uptake has resulted in LNP siRNA systems that can silence genes in hepatocytes at doses as low as 0.005 mg siRNA/kg body weight in mouse models. These systems have been shown to be highly effective clinically, with promising results for the treatment of hypercholesterolemia and transthyretin-induced amyloidosis among others. More LNP siRNA therapeutics, targeting different tissues and diseases, are expected to become available in the near future.
Identifiers
What OpenQuestion holds
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.