Gene therapy for primary ciliary dyskinesia (PCD) examines whether inherited defects in motile cilia can be addressed by restoring the missing or dysfunctional ciliary protein, correcting the underlying genetic defect, or supplying a transient therapeutic transcript to airway epithelial cells. Because PCD is genetically heterogeneous and primarily affects multiciliated respiratory cells, the field must connect molecular rescue with ciliary motion, mucus clearance, and long-term airway safety.
