ArticleBiomaterials2025
Ligand-free biodegradable poly(beta-amino ester) nanoparticles for targeted systemic delivery of mRNA to the lungs.
Article in Biomaterials, 2025. 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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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
23 citing papers in PubMed.
- Non-Viral CRISPR carriers: transient delivery with lasting effects.Drug delivery · 2026Review
- Shape-Programmable Particles from Photocurable Poly(β-Amino Esters) for Drug Delivery Applications.Advanced functional materials · 2026Article
- Delivery Systems for Therapeutic Genome Editing: Challenges, Innovations, and Future Perspectives.MedComm · 2026Review
- Degradable Nanocarriers Capable of Gene Delivery Derived from pH-Responsive Polyester: RROP Copolymerization Between Cyclic Ketene Acetals.Macromolecular rapid communications · 2026Article
- Remodeling the pathological airway: advanced nanotechnology for diagnostics and therapeutics in cystic fibrosis.Journal of nanobiotechnology · 2026Review
- Development of bicistronic plasmids and fusion proteins for clinical translation of tumor immune reprogramming.Molecular therapy. Advances · 2026Article
- Recent advances in the application of polymeric nanoparticles to the pulmonary delivery of mRNA.Nanomedicine (London, England) · 2026Review
- Review
- Base editing and nanoparticle transfection of airway cell types essential for treatment of cystic fibrosis.JCI insight · 2026Article
- Tissue-specific gene delivery approaches.Bioengineering & translational medicine · 2026Review
- Redox signaling in chronic airway diseases: pathogenic mechanisms and therapeutic implications.Frontiers in physiology · 2026Review
- Amino acid-based surfactants: sustainable synthesis and antimicrobial mechanisms.Beilstein journal of organic chemistry · 2026Review
- Near-Infrared Fluorescent Highly Branched Poly(β-Amino Ester)s Nanoparticles for Gene Delivery: In vivo Biodistribution and Safety Evaluation.International journal of nanomedicine · 2026Article
- Engineering Anti-Tumor Immunity: An Immunological Framework for mRNA Cancer Vaccines.Vaccines · 2025Review
- Base editing and nanoparticle transfection of airway cell types essential for treatment of cystic fibrosis.bioRxiv : the preprint server for biology · 2025Article
- A simple epoxide modification strategy to construct amino-functional poly(2-oxazoline) mRNA delivery vectors.Chemical science · 2025Article
- Recent advances in the nanoplatform-mediated delivery of gene editing technologies to the lungs.Nanomedicine (London, England) · 2025Article
- Recent Advances in mRNA Delivery Systems for Cancer Therapy.Advanced science (Weinheim, Baden-Wurttemberg, Germany) · 2025Review
- Cancer cell membrane-camouflaged pH-responsive nanoparticles for enhancing siRNA effect and synergistic anti-tumor therapy.Journal of nanobiotechnology · 2025Article
- Synthetic Polymers for Drug, Gene, and Vaccine Delivery.Polymer science & technology (Washington, D.C.) · 2025Review
Corrections and comments
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Authors and funding
5 authors.
Funding
Abstract
Non-viral nanoparticles (NPs) have seen heightened interest as a delivery method for a variety of clinically relevant nucleic acid cargoes in recent years. While much of the focus has been on lipid NPs, non-lipid NPs, including polymeric NPs, have the possibility of improved efficacy, safety, and targeting, especially to non-liver organs following systemic administration. A safe and effective systemic approach for intracellular delivery to the lungs could overcome limitations to intratracheal/intranasal delivery of NPs and improve clinical benefit for a range of diseases including cystic fibrosis. Here, engineered biodegradable poly (beta-amino ester) (PBAE) NPs are shown to facilitate efficient delivery of mRNA to primary human airway epithelial cells from both healthy donors and individuals with cystic fibrosis. Optimized NP formulations made with differentially endcapped PBAEs and systemically administered in vivo lead to high expression of mRNA within the lungs in BALB/c and C57 B/L mice without requiring a complex targeting ligand. High levels of mRNA-based gene editing were achieved in an Ai9 mouse model across bronchial, epithelial, and endothelial cell populations. No toxicity was observed either acutely or over time, including after multiple systemic administrations of the NPs. The non-lipid biodegradable PBAE NPs demonstrate high levels of transfection in both primary human airway epithelial cells and in vivo editing of lung cell types that are targets for numerous life-limiting diseases particularly single gene disorders such as cystic fibrosis and surfactant deficiencies.
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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.