Huljev Frković Sanda, Vičić Ana, Crkvenac Gornik Kristina, Kulišić Dinko, Stipoljev Feodora
Department of Paediatrics, Division for genetics and metabolism, University of Zagreb School of Medicine, University Hospital Centre Zagreb, Zagreb, Croatia.
Cytogenetic Laboratory, Department of Obstetrics and Gynecology, Clinical Hospital "Sveti Duh", Zagreb, Croatia.
Am J Med Genet A. 2022 Jun;188(6):1826-1830. doi: 10.1002/ajmg.a.62684. Epub 2022 Feb 15.
Primary cilia are a component of almost all vertebrate cells with a crucial role in sensing and transducing environmental signals during tissue development. Their dysfunction is known as ciliopathies and can manifest with a wide spectrum of clinical disorders. Overlapping features and molecular heterogeneity of ciliopathies make diagnoses distinctly challenging. In this group of diseases, tectonic genes, and their mutations play an important role. We present a first-trimester fetus with occipital encephalocele and OFD type IV caused by TCTN3 compound heterozygous pathogenic variants: c.1423_1429del (p.Arg475Serfs*10) and c.3G>A (initiator codon). A severe arm anomaly was described in our case, with two fingers along the atrophic forearm and polydactyly on other limbs. This could be a new phenotypic characteristic contributing to further understanding of TCTN3-related disorders as well as other tectonic proteins in ciliopathy spectrum diseases.
原发性纤毛是几乎所有脊椎动物细胞的组成部分,在组织发育过程中对感知和转导环境信号起着关键作用。它们的功能障碍被称为纤毛病,可表现为广泛的临床疾病。纤毛病的重叠特征和分子异质性使得诊断极具挑战性。在这组疾病中,结构基因及其突变起着重要作用。我们报告了一名孕早期胎儿,患有枕部脑膨出和由TCTN3复合杂合致病变异引起的IV型眼-脑-指综合征:c.1423_1429del(p.Arg475Serfs*10)和c.3G>A(起始密码子)。我们的病例中描述了一种严重的手臂异常,萎缩的前臂上有两根手指,其他肢体有多指畸形。这可能是一种新的表型特征,有助于进一步了解TCTN3相关疾病以及纤毛病谱系疾病中的其他结构蛋白。