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.
Department of Pediatrics, University of North Carolina School of Medicine, 101 Manning Drive, Chapel Hill, NC 27514, USA.
Int J Mol Sci. 2021 Jul 31;22(15):8272. doi: 10.3390/ijms22158272.
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.
原发性纤毛运动障碍(PCD)是一种罕见的遗传性疾病,影响游动纤毛,导致器官侧位缺陷、反复鼻窦-肺部感染、支气管扩张和严重肺部疾病。过去二十年来的研究揭示了临床表现的可变性,从轻到重的表型均有。已经出现了基因型和表型的关系。用于 PCD 的基因面板的日益普及继续重新定义这些基因型-表型关系,并揭示以前未被识别的更轻微的疾病形式。