Article

Targeted mRNA restoration of ciliary function in DNAI1-related primary ciliary dyskinesia: ex vivo rescue in patient-derived nasal spheroids – A pilot study

2026-05-28

Abstract excerpt

<h4>Rationale</h4> Primary Ciliary Dyskinesia (PCD) is a genetic disorder characterized by impaired ciliary function, defective mucociliary clearance, and progressive lung disease. Pathogenic variants in the DNAI1 gene are a well-known cause of PCD. Currently, no approved therapies address the underlying genetic defect. RCT1100 is an inhaled mRNA therapy encoding DNAI1 currently under clinical development. This...

Topics

Open a Topic to create a Post that cites this publication.

Identifiers and source

Literature Corpus work: cbbee076-87fa-522c-a6b9-4ae8c8df302f
DOI: 10.64898/2026.05.26.727937

Open publication

Related research

Semantic proximity does not establish scientific evidence.

Click a neighbor to travelStep 1 · 12 closest

Targeted mRNA restoration of ciliary function in DNAI1-related primary ciliary dyskinesia: ex vivo rescue in patient-derived nasal spheroids – A pilot studyDOI 10.64898/2026.05.26.727937Read

Personalized multi-assay profiling of respiratory motile ciliopathies and mRNA therapySemantic proximity Read

High-content screening for rare respiratory diseases: readthrough therapy in primary ciliary dyskinesiaSemantic proximity Read

Lentiviral Gene Delivery Rescues Ciliary Defects in Patient-Derived Airway Organoids from Primary Ciliary Dyskinesia.Semantic proximity Read

Inhaled delivery of a lipid nanoparticle encapsulated messenger RNA encoding a ciliary protein for the treatment of primary ciliary dyskinesia.Semantic proximity Read

Preclinical human models of primary ciliary dyskinesia.Semantic proximity Read

Current and Future Treatments in Primary Ciliary DyskinesiaSemantic proximity Read

Modelling of primary ciliary dyskinesia using patient-derived airway organoids.Semantic proximity Read

Patient-specific three-dimensional explant spheroids derived from human nasal airway epithelium: a simple methodological approach for ex vivo studies of primary ciliary dyskinesiaSemantic proximity Read

WS6 enables scalable <i>ex vivo</i> expansion and gene editing of human basal epithelial cellsSemantic proximity Read

Defining Functional Correction Thresholds in Primary Ciliary Dyskinesia for Effective Gene TherapiesSemantic proximity Read

Primary Ciliary Dyskinesia patient specific hiPSC-derived airway epithelium in Air Liquid Interface culture recapitulates disease specific phenotypes <i>in vitro</i>Semantic proximity Read

Development and Initial Characterization of Pigs with <i>DNAI1</i> Mutations and Primary Ciliary DyskinesiaSemantic proximity Read