Article

Defining Functional Correction Thresholds in Primary Ciliary Dyskinesia for Effective Gene Therapies

2026-02-20

Abstract excerpt

<h4>ABSTRACT</h4> <h4>Rationale</h4> Primary ciliary dyskinesia (PCD) is an inherited disorder characterized by defective motile cilia and impaired mucociliary clearance. Mutations in CCDC40 disrupt axonemal organization, resulting in dyskinetic or immotile cilia. While emerging therapies may restore function in only a subset of cells, the functional consequences of mixed populations of mutant and healthy cilia...

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Literature Corpus work: 534db7a3-906d-535f-8e16-013cded58f6d
DOI: 10.64898/2026.02.19.706855

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