Department of Pathology, Dalhousie University, Halifax, Nova Scotia, Canada.
Nat Genet. 2011 Feb 27;43(4):360-4. doi: 10.1038/ng.777.
Meier-Gorlin syndrome is a rare autosomal recessive genetic condition whose primary clinical hallmarks include small stature, small external ears and small or absent patellae. Using marker-assisted mapping in multiple families from a founder population and traditional coding exon sequencing of positional candidate genes, we identified three different mutations in the gene encoding ORC4, a component of the eukaryotic origin recognition complex, in five individuals with Meier-Gorlin syndrome. In two such individuals that were negative for mutations in ORC4, we found potential mutations in ORC1 and CDT1, two other genes involved in origin recognition. ORC4 is well conserved in eukaryotes, and the yeast equivalent of the human ORC4 missense mutation was shown to be pathogenic in functional assays of cell growth. This is the first report, to our knowledge, of a germline mutation in any gene of the origin recognition complex in a vertebrate organism.
Meier-Gorlin 综合征是一种罕见的常染色体隐性遗传疾病,其主要临床特征包括身材矮小、小耳朵和髌骨小或缺失。通过对来自一个奠基人群体的多个家族进行标记辅助图谱绘制和定位候选基因的传统编码外显子测序,我们在五名患有 Meier-Gorlin 综合征的个体中鉴定出编码真核起始识别复合物组成部分 ORC4 的基因中的三个不同突变。在两个 ORC4 基因突变阴性的个体中,我们发现了 ORC1 和 CDT1 这两个参与起始识别的其他基因的潜在突变。ORC4 在真核生物中高度保守,人类 ORC4 错义突变的酵母等效物在细胞生长的功能测定中被证明是致病性的。据我们所知,这是第一个在脊椎动物生物中发现起始识别复合物任何基因的种系突变的报告。