ReviewMolecular therapy. Methods & clinical development2025
Rational design of lipid nanoparticles for enabling gene therapies.
Review in Molecular therapy. Methods & clinical development, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 19 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
19 citing papers in PubMed.
- Gene therapy for hereditary hematological disorders: From clinical breakthroughs to future horizons.Molecular therapy. Nucleic acids · 2026Review
- Overcoming hepatic tropism: Precision engineering of lipid nanoparticles for extrahepatic RNA delivery.Materials today. Bio · 2026Review
- Dual pKa Lipid Nanoparticles for Lung-tropic mRNA Delivery and pH-Programmed Endosomal Escape.Advanced science (Weinheim, Baden-Wurttemberg, Germany) · 2026Article
- Membrane environment sets the functional pKBiophysical journal · 2026Article
- Biomaterial-Integrated Electroporation for Therapeutic Delivery: From Gene Editing to Tumor Ablation and Immune Modulation.Small (Weinheim an der Bergstrasse, Germany) · 2026Review
- Review
- From Morphology to Mechanism: Cryo-Electron Microscopy Insights into Lipid Nanoparticles for RNA Delivery.ACS nano · 2026Review
- In-Depth Characterization of PEGylated Liposomes: Using AF4 and HPLC-CAD in Tandem as a Strategy for Composition Assessment and Postinsertion Optimization.Analytical chemistry · 2026Article
- Considerations for early life genetic therapies in cystic fibrosis.American journal of physiology. Lung cellular and molecular physiology · 2026Review
- LNP-based delivery of a Toll-like receptor 9 agonist elicits potent adjuvant effects and antitumor immunity.NPJ vaccines · 2026Article
- Recent advances in lipid and biomimetic nanocarriers for nucleic acid delivery in glioblastoma.Discover oncology · 2026Review
- Lipid nanoparticles for cell and gene therapy.Molecular therapy. Advances · 2026Article
- RNA-Based Therapeutic Strategies in Multiple Myeloma: From Molecular Targets to Delivery and Clinical Translation.International journal of molecular sciences · 2026Review
- Clinical translation of CRISPR-Cas9 therapeutics in cancer and inherited genetic disorders.Frontiers in genome editing · 2026Review
- Protein-based custom-designed molecular nanotraps for biomedical applications.Beilstein journal of nanotechnology · 2026Article
- High-Resolution Characterization of Protein-Conjugated, mRNA-Loaded Lipid Nanoparticles by Analytical Ultracentrifugation.Advanced functional materials · 2025Article
- Translational Advances in Lipid Nanoparticle Drug Delivery Systems for Cancer Therapy: Current Status and Future Horizons.Pharmaceutics · 2025Review
- Radiosensitization by Docetaxel Prodrug-Loaded Lipid Nanoparticles in Pancreatic Cancer Xenografts.Nanomaterials (Basel, Switzerland) · 2025Article
- Emerging Approaches for the Discovery of Lipid-Based RNA Delivery Systems.Pharmaceutics · 2025Review
Corrections and comments
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Authors and funding
6 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Lipid nanoparticle (LNP) technology is increasingly enabling RNA-based gene therapies that can potentially be used to treat most diseases. Further, these LNP RNA therapeutics can be designed and manufactured in a matter of weeks, allowing personalized medicines that can be produced in a time frame relevant to individuals suffering from terminal diseases. Here, we focus on the rational design principles that have successfully enabled LNP small interfering RNA (siRNA) formulations to silence pathogenic genes in the liver and LNP mRNA formulations to express therapeutic proteins for vaccines and gene therapies. These principles have evolved from over 50 years of research into the physical properties and functional roles of lipids in membranes as well as experience gained developing LNP systems for delivery of small molecule drugs. It is expected that these rational design principles will be successful in enabling most forms of gene therapies.
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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.