ArticleProceedings of the National Academy of Sciences of the United States of America2025
Inhaled
Article in Proceedings of the National Academy of Sciences of the United States of America, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 12 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
12 citing papers in PubMed.
- Towards mRNA therapeutics 2.0.Nature reviews. Drug discovery · 2026Review
- Diagnosing primary ciliary dyskinesia in Australian adults: 10 years of testing.Internal medicine journal · 2026Article
- mRNA therapy improves the composition and motility in CCDC40-deficient cilia in vitro and in vivo.American journal of respiratory cell and molecular biology · 2026Article
- Airway Clearance and Pediatric Pulmonary Rehabilitation in Primary Ciliary Dyskinesia: A Clinical Framework for Children and Adolescents.Journal of clinical medicine · 2026Review
- Two Classes of Protein Therapeutics: Why Dose-Response Architecture Defines the Boundary of mRNA Medicines.Pharmaceutics · 2026Review
- Target-Product and Translational Design Principles for Inhalable RNA Nanomedicines.Pharmaceutics · 2026Review
- Preclinical human models of primary ciliary dyskinesia.European respiratory review : an official journal of the European Respiratory Society · 2026Review
- Investigating the impact of poly(beta amino) ester-mediated FOXJ1 mRNA delivery on differentiation of primary human bronchial epithelial cells.Biology open · 2026Article
- Noncoding DNA Variants in Primary Ciliary Dyskinesia: Another Piece of the Diagnostic Puzzle.American journal of respiratory and critical care medicine · 2025Article
- Lipid nanoparticle-encapsulated Dnai1 mRNA rescues ciliary activity in primary ciliary dyskinesia mouse cell models.Journal of cell science · 2025Article
- Primary Ciliary Dyskinesia-Current Diagnostic and Therapeutic Approach.Journal of clinical medicine · 2025Review
- A stem cell-based platform for functional analysis of genetic variants in lung disease.bioRxiv : the preprint server for biology · 2025Article
Corrections and comments
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Authors and funding
19 authors.
Funding
Abstract
Primary ciliary dyskinesia (PCD) is an autosomal recessive disorder caused by mutations in one of at least 50 different genes that encode proteins involved in the biogenesis, structure, or function of motile cilia. Genetically inherited defects in motile cilia cause PCD, a debilitating respiratory disease for which there is no approved therapy. The dynein axonemal intermediate chain 1 (DNAI1) protein is a key structural element of the ciliary outer dynein arm (ODA) critical for normal ciliary activity and subsequent clearance of mucus from the conducting airways in humans. Loss-of-function mutations in DNAI1 account for up to 10% of all PCD cases, with functional abnormalities in patients presenting at or near birth and leading to a life-long course of disability, including progressive loss of lung function and bronchiectasis by adulthood. This underscores the significant unmet need for disease-modifying treatments that restore ciliary activity and mucociliary clearance in PCD patients. In this work, we demonstrate that lipid nanoparticle (LNP)-formulated human
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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.