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对35名患有过度生长综合征(包括双等位基因SUZ12变异)儿童的病因及随访结果进行调查。

An investigation of the etiology and follow-up findings in 35 children with overgrowth syndromes, including biallelic SUZ12 variant.

作者信息

Yüksel Ülker Aylin, Uludağ Alkaya Dilek, Çağlayan Ahmet Okay, Usluer Esra, Aykut Ayça, Aslanger Ayça, Vural Mehmet, Tüysüz Beyhan

机构信息

Department of Pediatric Genetics, Cerrahpasa Medical Faculty, Istanbul University-Cerrahpasa, Istanbul, Turkey.

Departments of Neurosurgery, Neurobiology and Genetics, Yale School of Medicine, New Haven, Connecticut, USA.

出版信息

Am J Med Genet A. 2023 Jun;191(6):1530-1545. doi: 10.1002/ajmg.a.63180. Epub 2023 Mar 15.

DOI:10.1002/ajmg.a.63180
PMID:36919607
Abstract

Overgrowth-intellectual disability (OGID) syndromes are clinically and genetically heterogeneous group of disorders. The aim of this study was to examine the molecular etiology and long-term follow-up findings of Turkish OGID cohort. Thirty-five children with OGID were included in the study. Single gene sequencing, clinical exome analysis, chromosomal microarray analysis and whole exome sequencing were performed. Five pathogenic copy number variants were detected in the patients; three of them located on chromosome 5q35.2 (encompassing NSD1), others on 9q22.3 and 22q13.31. In 19 of 35 patients; we identified pathogenic variants in OGID genes associated with epigenetic regulation, NSD1 (n = 15), HIST1H1E (n = 1), SETD1B (n = 1), and SUZ12 (n = 2). The pathogenic variants in PIK3CA (n = 2), ABCC9 (n = 1), GPC4 (n = 2), FIBP (n = 1), and TMEM94 (n = 1) which had a role in other growth pathways were detected in seven patients. The diagnostic yield was 31/35(88%). Twelve pathogenic variants were novel. The common facial feature of the patients was prominent forehead. The patients with Sotos syndrome were observed to have milder intellectual disability than patients with other OGID syndromes. In conclusion, this study showed, for the first time, that biallelic variants of SUZ12 caused Imagawa-Matsumoto syndrome, monoallelic variants in SETDIB resulted in OGID. Besides expanded the phenotypes of very rare OGID syndromes caused by FIBP and TMEM94.

摘要

过度生长-智力障碍(OGID)综合征是一组临床和遗传异质性疾病。本研究的目的是探讨土耳其OGID队列的分子病因及长期随访结果。35名OGID患儿纳入本研究。进行了单基因测序、临床外显子组分析、染色体微阵列分析和全外显子组测序。在患者中检测到5个致病性拷贝数变异;其中3个位于5号染色体q35.2(包含NSD1),其他分别位于9号染色体q22.3和22号染色体q13.31。35例患者中有19例;我们在与表观遗传调控相关的OGID基因中鉴定出致病性变异,NSD1(n = 15)、HIST1H1E(n = 1)、SETD1B(n = 1)和SUZ12(n = 2)。在7例患者中检测到PIK3CA(n = 2)、ABCC9(n = 1)、GPC4(n = 2)、FIBP(n = 1)和TMEM94(n = 1)中在其他生长途径中起作用的致病性变异。诊断率为31/35(88%)。12个致病性变异是新发现的。患者常见的面部特征是前额突出。观察到索托斯综合征患者的智力障碍比其他OGID综合征患者轻。总之,本研究首次表明,SUZ12的双等位基因变异导致今川-松本综合征,SETD1B的单等位基因变异导致OGID。此外,还扩展了由FIBP和TMEM94引起的非常罕见的OGID综合征的表型。

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