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两名 Temple 综合征新患者存在 UPD(14)mat 和 UPD(14)mat 并伴有 14 号染色体亚端粒标记染色体嵌合体。

UPD(14)mat and UPD(14)mat in concomitance with mosaic small supernumerary marker chromosome 14 in two new patients with Temple syndrome.

机构信息

Departamento de Genética Humana, Instituto Nacional de Pediatría. Ciudad de México, Mexico.

Laboratorio de Genética y Cáncer, Departamento de Genética Humana, Instituto Nacional de Pediatría. Ciudad de México, 04530, Mexico.

出版信息

Eur J Med Genet. 2021 May;64(5):104199. doi: 10.1016/j.ejmg.2021.104199. Epub 2021 Mar 18.

Abstract

Temple syndrome (TS14) can be originated by maternal uniparental disomy (UPD(14)mat), paternal deletion, or epimutation, leading to disturbances in 14q32.2 imprinted region. The most frequent phenotypic manifestations are prenatal and postnatal growth failure, hypotonia, developmental delay, small hands/feet, precocious puberty, and truncal obesity. However, the diagnosis can be challenging due to the clinical overlap with other imprinting disorders such as Silver-Russell or Prader-Willi syndromes. Although rare, TS14 has been also reported in patients with concomitant UPD(14)mat and mosaic trisomy 14. In the present report, the clinical and genetic profiles of two new patients with TS14 are described. SNParray and MS-MLPA, allowed the determination of segmental UPD(14)mat and the hypomethylation of MEG3 gene. Additionally, in one of our patients we also observed by cytogenetics a small supernumerary marker chromosome that led to partial trisomy 14 in mosaic. Only few patients with concomitant UPD(14)mat and mosaic partial trisomy 14 have been reported. Our patients share cardinal TS14 phenotypic features that are associated to the genetic abnormalities detected; however, we also observed some clinical features such as fatty liver disease that had not previously been reported as part of this syndrome. The detailed clinical, cytogenetical and molecular description of these two new patients, contributes to a more accurately delineation of this syndrome.

摘要

泰-萨克斯二氏综合征(TS14)可由母体单亲二体(UPD(14)mat)、父源缺失或印迹基因异常导致 14q32.2 印迹区域功能紊乱引起。最常见的表型特征为产前和产后生长发育迟缓、肌张力低下、发育迟缓、手脚短小、性早熟和躯干性肥胖。然而,由于其与其他印迹疾病(如 Silver-Russell 或 Prader-Willi 综合征)存在临床重叠,因此诊断可能具有挑战性。尽管罕见,但 TS14 也已在伴有 UPD(14)mat 和镶嵌性三体 14 的患者中报道。本研究报道了两名新的 TS14 患者的临床和遗传学特征。通过 SNP 阵列和 MS-MLPA,确定了部分 UPD(14)mat 和 MEG3 基因的低甲基化。此外,在我们的一名患者中,我们还通过细胞遗传学观察到一个小的额外标记染色体,导致镶嵌性部分三体 14。仅有少数伴有 UPD(14)mat 和镶嵌性部分三体 14 的患者被报道。我们的患者具有 TS14 的主要表型特征,这些特征与检测到的遗传异常相关;然而,我们还观察到一些以前未被报道为该综合征一部分的临床特征,如脂肪肝疾病。这两名新患者的详细临床、细胞遗传学和分子描述有助于更准确地描绘该综合征。

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