Article
Preclinical human models of primary ciliary dyskinesia.
European respiratory review : an official journal of the European Respiratory Society - 1 Jul 2026
Ong Jonathan W Y, Jackson Claire L, Arbi Marina, Hart Stephen L, Mill Pleasantine, Paff Tamara, Rubbo Bruna, Shahaj Eriomina, Shoemark Amelia, Lucas Jane S
Abstract excerpt
Primary ciliary dyskinesia (PCD) is a genetically heterogeneous disorder caused by defective motile cilia, resulting in impaired mucociliary clearance, chronic respiratory disease, laterality defects and subfertility. Currently, no disease-modifying treatments exist. Targeting PCD at its root cause requires emerging genetic therapies, such as small molecules, oligonucleotides, mRNA therapy, gene replacement and...
Read the complete abstract on PubMedTopics
Share this publication in a Topic to start or enrich a Post.
