Genetic Medicine division, Diagnostic Department, Hôpitaux Universitaires de Genève, Genève (CH), Switzerland.
Pediatric Specialties division, Department of Women, Children and Adolescents, Hôpitaux Universitaires de Genève, Genève (CH), Switzerland.
Clin Genet. 2021 Sep;100(3):329-333. doi: 10.1111/cge.14004. Epub 2021 Jun 14.
Arthrogryposis describes the presence of multiple joint-contractures. Clinical severity of this phenotype is variable, and more than 400 causative genes have been proposed. Among these, ERGIC1 is a recently reported candidate encoding a putative transmembrane protein of the ER-Golgi interface. Two homozygous missense variants have been reported in patients with relatively mild non-syndromic arthrogryposis. In a consanguineous family with two affected siblings presenting congenital arthrogryposis and some facial dysmorphism we performed prenatal array-CGH, postnatal targeted exome and genome sequencing. Genome sequencing identified a homozygous 22.6 Kb deletion encompassing the promoter and first exon of ERGIC1. mRNA quantification showed the complete absence of ERGIC1 expression in the two affected siblings and a decrease in heterozygous parents. Our observations validate the pathogenic role of ERGIC1 in congenital arthrogryposis and demonstrate that complete loss of function causes a relatively mild phenotype. These findings will contribute to improve genetic counseling of ERGIC1 mutations.
关节挛缩症是指多个关节出现挛缩。该表型的临床严重程度存在差异,据报道已有超过 400 个致病基因。其中,ERGIC1 是一种新报道的候选基因,其编码内质网-高尔基体界面的假定跨膜蛋白。已有报道称,两名患有相对较轻的非综合征性关节挛缩症的患者携带有两个纯合错义变异。在一个有两个受影响的兄弟姐妹的近亲家庭中,他们患有先天性关节挛缩症和一些面部畸形,我们进行了产前 array-CGH、产后靶向外显子组和全基因组测序。基因组测序发现了一个纯合的 22.6kb 缺失,包含 ERGIC1 的启动子和第一外显子。mRNA 定量显示,两个受影响的兄弟姐妹中 ERGIC1 的表达完全缺失,杂合父母的表达减少。我们的观察结果证实了 ERGIC1 在先天性关节挛缩症中的致病性作用,并表明完全丧失功能会导致相对较轻的表型。这些发现将有助于改善 ERGIC1 突变的遗传咨询。