ArticleMolecular therapy : the journal of the American Society of Gene Therapy2018
Lipid Nanoparticle-Delivered Chemically Modified mRNA Restores Chloride Secretion in Cystic Fibrosis.
Article in Molecular therapy : the journal of the American Society of Gene Therapy, 2018. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 133 papers, 2 of them syntheses that pooled it.
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
133 citing papers in PubMed, 2 syntheses or guidelines pooled it, 259 citations in OpenAlex.
- A systematic review and meta-analysis of the treatment modalities available for children afflicted from cystic fibrosis.BMC pediatrics · 2025Pooled it
- A systematic review of animal and human data comparing the nasal potential difference test between cystic fibrosis and control.Scientific reports · 2024Pooled it
- Elastic Fibers in the Pathogenesis of Cystic Fibrosis: Mechanisms, Consequences, and Therapeutic Implications.Life (Basel, Switzerland) · 2026Review
- Precision nanomedicine for pulmonary diseases: from molecular targeting to clinical translation.Signal transduction and targeted therapy · 2026Review
- Combining Gene Therapy with Current Modulator Treatments for Cystic Fibrosis: A Promising Area of Research.Pharmaceutics · 2026Review
- Muscle-targeting LNP vaccines enable potent immune responses against varicella zoster virus.Drug delivery and translational research · 2026Article
- mRNA Lipid Nanoparticles for Cell Engineering in Vivo and in Vitro: Current Applications and Future Directions.MedComm · 2026Review
- Inhalable gene and RNA therapy for cystic fibrosis: perspectives and progress in clinical development.Nanomedicine (London, England) · 2026Review
- Transamniotic Fetal Delivery of Cystic Fibrosis (CF) Transmembrane Conductance Regulator (CFTR) mRNA.FASEB bioAdvances · 2026Article
- 'Tripod-like' lung-targeting (LuT) lipids for highly efficient and selective LNPs for gene delivery and editing.Nature biomedical engineering · 2026Article
- Lung-targeted RNA delivery systems: strategies and therapeutic applications.Journal of nanobiotechnology · 2026Review
- Interleukin 6 Drives Durable T Cell-Mediated Immunity to Pancreatic Cancer.Cellular and molecular gastroenterology and hepatology · 2026Article
- mRNA therapeutics: Transforming medicine through innovation in design, delivery, and disease treatment.Molecular therapy. Nucleic acids · 2025Review
- Advances in Lipid-Polymer Hybrid Nanoparticles: Design Strategies, Functionalization, Oncological and Non-Oncological Clinical Prospects.Pharmaceuticals (Basel, Switzerland) · 2025Review
- Advancing Lung Cancer Treatment: mRNA Therapeutics and Delivery Strategies.Nano letters · 2025Review
- Nanotechnology and CRISPR/Cas-Mediated Gene Therapy Strategies: Potential Role for Treating Genetic Disorders.Molecular biotechnology · 2025Review
- Nanoparticle delivery of VEGF-B mRNA promotes T cell infiltration within tumor and triggers robust antitumor immunity.Molecular therapy. Nucleic acids · 2025Article
- Freeze-Drying of mRNA-LNPs Vaccines: A Review.Vaccines · 2025Review
- Exploring the Potential and Advancements of Circular RNA Therapeutics.Exploration (Beijing, China) · 2025Review
- RNA lipid nanoparticles stabilized during nebulization through excipient selection.Nanoscale advances · 2025Article
73 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
7 authors at 2 institutions in 1 country.
Funding
Abstract
The promise of gene therapy for the treatment of cystic fibrosis has yet to be fully clinically realized despite years of effort toward correcting the underlying genetic defect in the cystic fibrosis transmembrane conductance regulator (CFTR). mRNA therapy via nanoparticle delivery represents a powerful technology for the transfer of genetic material to cells with large, widespread populations, such as airway epithelia. We deployed a clinically relevant lipid-based nanoparticle (LNP) for packaging and delivery of large chemically modified CFTR mRNA (cmCFTR) to patient-derived bronchial epithelial cells, resulting in an increase in membrane-localized CFTR and rescue of its primary function as a chloride channel. Furthermore, nasal application of LNP-cmCFTR restored CFTR-mediated chloride secretion to conductive airway epithelia in CFTR knockout mice for at least 14 days. On day 3 post-transfection, CFTR activity peaked, recovering up to 55% of the net chloride efflux characteristic of healthy mice. This magnitude of response is superior to liposomal CFTR DNA delivery and is comparable with outcomes observed in the currently approved drug ivacaftor. LNP-cmRNA-based systems represent a powerful platform technology for correction of cystic fibrosis and other monogenic disorders.
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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.