Emerging Genotype-Phenotype Relationships in Primary Ciliary Dyskinesia
- Int J Mol Sci. 2021 Jul 31;22(15):8272. doi: 10.3390/ijms22158272.
- 1. Department of Pediatrics, Division of Allergy and Pulmonary Medicine, Campus Box 8116, Washington University School of Medicine, 660 South Euclid Avenue, St. Louis, MO 63110, USA.
- 2. Department of Pediatrics, University of North Carolina School of Medicine, 101 Manning Drive, Chapel Hill, NC 27514, USA.
Primary ciliary dyskinesia (PCD) is a rare inherited condition affecting motile cilia and leading to organ laterality defects, recurrent sino-pulmonary infections, bronchiectasis, and severe lung disease. Research over the past twenty years has revealed variability in clinical presentations, ranging from mild to more severe phenotypes. Genotype and phenotype relationships have emerged. The increasing availability of genetic panels for PCD continue to redefine these genotype-phenotype relationships and reveal milder forms of disease that had previously gone unrecognized.