Defining functional correction thresholds in primary ciliary dyskinesia for effective gene therapies

Extract Mucociliary clearance depends on the coordinated activity of motile cilia across an organised airway epithelium, and disruption of this system leads to chronic airway disease [1, 2]. In primary ciliary dyskinesia (PCD), diverse structural and functional ciliary defects, including absent cilia, axonemal abnormalities or impaired coordination, produce highly variable clinical phenotypes [3–6].