Targeted mRNA restoration of ciliary function in DNAI1-related primary ciliary dyskinesia: ex vivo rescue in patient-derived nasal spheroids, A pilot study
Caroline Marie Torp Nygaard, Cristian Herrera-Cid, Laura Nedergaard, Stine Green Johansen, J G Matthews, Jessica A. Couch, Tavs Qvist, Kim G Nielsen, Søren T. Christensen, June Kehlet Marthin
This study evaluates an mRNA therapy for DNAI1-related PCD using patient-derived nasal spheroids, showing increased ciliary function and spheroid motility after treatment.
bioRxiv · May 28, 2026 · PreprintPhysiology