PhysiologybioRxivPreprint
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…·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.
DOI 10.64898/2026.05.26.727937