CCDC39 对于人类和犬类的内臂动力蛋白和动力蛋白调节复合物的组装以及正常的纤毛运动是必需的。
CCDC39 is required for assembly of inner dynein arms and the dynein regulatory complex and for normal ciliary motility in humans and dogs.
机构信息
Unit of Animal Genomics, Groupe Interdisciplinaire de Genomique Appliquee-Recherche (GIGA-R) and Faculty of Veterinary Medicine, University of Liège (B34), Liège, Belgium.
出版信息
Nat Genet. 2011 Jan;43(1):72-8. doi: 10.1038/ng.726. Epub 2010 Dec 5.
Primary ciliary dyskinesia (PCD) is an inherited disorder characterized by recurrent infections of the upper and lower respiratory tract, reduced fertility in males and situs inversus in about 50% of affected individuals (Kartagener syndrome). It is caused by motility defects in the respiratory cilia that are responsible for airway clearance, the flagella that propel sperm cells and the nodal monocilia that determine left-right asymmetry. Recessive mutations that cause PCD have been identified in genes encoding components of the outer dynein arms, radial spokes and cytoplasmic pre-assembly factors of axonemal dyneins, but these mutations account for only about 50% of cases of PCD. We exploited the unique properties of dog populations to positionally clone a new PCD gene, CCDC39. We found that loss-of-function mutations in the human ortholog underlie a substantial fraction of PCD cases with axonemal disorganization and abnormal ciliary beating. Functional analyses indicated that CCDC39 localizes to ciliary axonemes and is essential for assembly of inner dynein arms and the dynein regulatory complex.
原发性纤毛运动障碍(PCD)是一种遗传性疾病,其特征是上呼吸道和下呼吸道反复感染、男性生育能力降低以及约 50%的受影响个体出现位置反转(卡塔格纳综合征)。它是由负责气道清除、推动精子细胞的鞭毛和决定左右不对称的节点纤毛的呼吸纤毛运动缺陷引起的。已经在编码外动力蛋白臂、辐条和轴丝动力蛋白细胞质预组装因子的基因中鉴定出导致 PCD 的隐性突变,但这些突变仅占 PCD 病例的约 50%。我们利用犬种群的独特特性,对一个新的 PCD 基因 CCDC39 进行了定位克隆。我们发现,人类同源物的功能丧失突变是轴丝结构紊乱和纤毛运动异常的 PCD 病例的重要基础。功能分析表明,CCDC39 定位于纤毛轴丝,对于内动力蛋白臂和动力蛋白调节复合物的组装是必需的。
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