Institute of Medical and Human Genetics, Charité-Universitätsmedizin Berlin, Berlin, Germany.
Cologne Center for Genomics, University of Cologne, University Hospital Cologne, Cologne, Germany.
Hum Genet. 2022 Nov;141(11):1785-1794. doi: 10.1007/s00439-022-02461-w. Epub 2022 May 10.
The evolutionary conserved Polo-like kinase 4 (PLK4) is essential for centriole duplication, spindle assembly, and de novo centriole formation. In man, homozygous mutations in PLK4 lead to primary microcephaly, altered PLK4 expression is associated with aneuploidy in human embryos. Here, we report on a consanguineous four-generation family with 8 affected individuals compound heterozygous for a novel missense variant, c.881 T > G, and a deletion of the PLK4 gene. The clinical phenotype of the adult patients is mild compared to individuals with previously described PLK4 mutations. One individual was homozygous for the variant c.881G and phenotypically unaffected. The deletion was inherited by 14 of 16 offspring and thus exhibits transmission ratio distortion (TRD). Moreover, based on the already published families with PLK4 mutations, it could be shown that due to the preferential transmission of the mutant alleles, the number of affected offspring is significantly increased. It is assumed that reduced expression of PLK4 decreases the intrinsically high error rate of the first cell divisions after fertilization, increases the number of viable embryos and thus leads to preferential transmission of the deleted/mutated alleles.
进化上保守的 Polo 样激酶 4(PLK4)对于中心体复制、纺锤体组装和新中心体形成是必不可少的。在人类中,PLK4 的纯合突变导致原发性小头畸形,PLK4 表达的改变与人类胚胎的非整倍体有关。在这里,我们报告了一个四代同堂的近亲家庭,有 8 名受影响的个体复合杂合了一种新的错义变异 c.881T>G,以及 PLK4 基因缺失。与先前描述的 PLK4 突变个体相比,成年患者的临床表型较轻。有一个个体为 c.881G 变异的纯合子,但表型不受影响。该缺失由 16 个后代中的 14 个遗传,因此表现出传递率偏倚(TRD)。此外,根据已经发表的带有 PLK4 突变的家族,可以表明由于突变等位基因的优先传递,受影响的后代数量显著增加。据推测,PLK4 的表达减少降低了受精后第一次细胞分裂的固有高错误率,增加了存活胚胎的数量,从而导致缺失/突变等位基因的优先传递。