Medical Genetics Unit, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan, Italy.
Laboratorio di Genetica Medica, ASST Papa Giovanni XXIII, Bergamo, Italy.
Am J Med Genet A. 2023 Jun;191(6):1586-1592. doi: 10.1002/ajmg.a.63164. Epub 2023 Feb 26.
Cornelia de Lange syndrome (CdLS) is a rare multisystem congenital neurodevelopmental disorder (NDD) characterized by distinctive facial anomalies, short stature, developmental delay, hirsutism, gastrointestinal abnormalities and upper limb reduction defects. CdLS syndrome is associated with causative variants in genes encoding for the cohesin complex, a cellular machinery involved in chromatid pairing, DNA repair and gene-expression regulation. In this report, we describe a familial case of a syndromic presentation in a 4-year-old patient (P1) and in his mother (P2). Trio-based Whole Exome Sequencing (WES) performed on P1 was first negative. Since his phenotypic evolution during the follow-up was reminiscent of the CdLS spectrum, a reanalysis of WES data, focused on CdLS-related genes, was requested. Although no alterations in those genes was detected, we identified the likely pathogenetic variant c.40G > A (p.Glu14Lys) in the PHIP gene, in the meanwhile associated with Chung-Jansen syndrome. Reverse phenotyping carried out in both patients confirmed the molecular diagnosis. CHUJANS belongs to NDDs, featuring developmental delay, mild-to-moderate intellectual disability, behavioral problems, obesity and facial dysmorphisms. Moreover, as here described, CHUJANS shows a significant overlap with the CdLS spectrum, with specific regard to facial gestalt. On the basis of our findings, we suggest to include PHIP among genes routinely analyzed in patients belonging to the CdLS spectrum.
康尼氏综合征(CdLS)是一种罕见的多系统先天性神经发育障碍(NDD),其特征为独特的面部异常、身材矮小、发育迟缓、多毛症、胃肠道异常和上肢减少缺陷。CdLS 综合征与编码黏合复合物的基因中的致病变异有关,该复合物是一种参与染色单体配对、DNA 修复和基因表达调控的细胞机制。在本报告中,我们描述了一个 4 岁患者(P1)及其母亲(P2)的综合征表现家族病例。对 P1 进行的基于三亲体的全外显子组测序(WES)最初为阴性。由于他在随访期间的表型演变类似于 CdLS 谱,因此要求对 WES 数据进行重新分析,重点关注与 CdLS 相关的基因。尽管在这些基因中未检测到改变,但我们在 PHIP 基因中鉴定出可能的致病性变异 c.40G > A(p.Glu14Lys),同时该变异与 Chung-Jansen 综合征相关。在两名患者中进行的反向表型分析证实了分子诊断。CHUJANS 属于 NDD,表现为发育迟缓、轻度至中度智力残疾、行为问题、肥胖和面部畸形。此外,如本文所述,CHUJANS 与 CdLS 谱有明显重叠,特别是在面部整体特征方面。基于我们的发现,我们建议将 PHIP 基因纳入到 CdLS 谱患者中常规分析的基因中。