ArticleNature nanotechnology2019
Self-assembled peptide-poloxamine nanoparticles enable in vitro and in vivo genome restoration for cystic fibrosis.
Article in Nature nanotechnology, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 55 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.
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Who cites it
55 citing papers in PubMed.
- Precision nanomedicine for pulmonary diseases: from molecular targeting to clinical translation.Signal transduction and targeted therapy · 2026Review
- Bacterial mRNA Vaccines: Programming Immunity Against Antimicrobial Resistance.Drug development research · 2026Review
- Remodeling the pathological airway: advanced nanotechnology for diagnostics and therapeutics in cystic fibrosis.Journal of nanobiotechnology · 2026Review
- Inhalable gene and RNA therapy for cystic fibrosis: perspectives and progress in clinical development.Nanomedicine (London, England) · 2026Review
- Harnessing semen-derived exosomes for noninvasive fundus drug delivery: A paradigm for exosome-based ocular fundus therapeutics.Science advances · 2026Article
- Aerosol Delivery of Polyelectrolyte Surfactant-Antimicrobial Nanoparticles to the Lungs.Pharmaceutical research · 2026Article
- Lung-targeted RNA delivery systems: strategies and therapeutic applications.Journal of nanobiotechnology · 2026Review
- Structure-guided design of nucleosome-inspired nanoparticles for overcoming pulmonary barriers in fibrotic lung gene therapy.Science advances · 2025Article
- Virus-inspired lipopeptide-derived nucleic acid delivery to cartilage for osteoarthritis therapy.Nature communications · 2025Article
- Review
- Airway-applied mRNA vaccine needs tailored sequence design and high standard purification that removes devastating dsRNA contaminant.Molecular therapy : the journal of the American Society of Gene Therapy · 2025Article
- Antimicrobial Loaded Graft-Copolymer Nanoparticles for Treatment ofbioRxiv : the preprint server for biology · 2025Article
- Recent Advances in Therapeutic Peptides: Innovations and Applications in Treating Infections and Diseases.ACS omega · 2025Review
- Inhaled non-viral delivery systems for RNA therapeutics.Acta pharmaceutica Sinica. B · 2025Review
- Recent strategies for enhanced delivery of mRNA to the lungs.Nanomedicine (London, England) · 2025Review
- Hydrogen-induced disruption of the airway mucus barrier enhances nebulized RNA delivery to reverse pulmonary fibrosis.Science advances · 2025Article
- Peptide-Bound Aflibercept Eye Drops for Treatment of Neovascular Age-Related Macular Degeneration in Nonhuman Primates.Advanced science (Weinheim, Baden-Wurttemberg, Germany) · 2025Article
- Hot Melt Extrusion as Continuous Manufacturing Technique to Produce Bilayer Films Loaded with Paracetamol or Lactase.Pharmaceuticals (Basel, Switzerland) · 2025Article
- Ionizable polymeric micelles (IPMs) for efficient siRNA delivery.Nature communications · 2025Article
- Gene Therapy: Towards a New Era of Medicine.AAPS PharmSciTech · 2024Review
Corrections and comments
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Authors and funding
18 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Developing safe and efficient non-viral delivery systems remains a major challenge for in vivo applications of gene therapy, especially in cystic fibrosis. Unlike conventional cationic polymers or lipids, the emerging poloxamine-based copolymers display promising in vivo gene delivery capabilities. However, poloxamines are invalid for in vitro applications and their in vivo transfection efficiency is still low compared with viral vectors. Here, we show that peptides developed by modular design approaches can spontaneously form compact and monodisperse nanoparticles with poloxamines and nucleic acids via self-assembly. Both messenger RNA and plasmid DNA expression mediated by peptide-poloxamine nanoparticles are greatly boosted in vitro and in the lungs of cystic fibrosis mice with negligible toxicity. Peptide-poloxamine nanoparticles containing integrating vectors enable successful in vitro and in vivo long-term restoration of cystic fibrosis transmembrane conductance regulator deficiency with a safe integration profile. Our dataset provides a new framework for designing non-viral gene delivery systems qualified for in vivo genetic modifications.
Indexed as
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30692673What 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.