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苏丹两家族性先天性甲状腺功能减退症患者致因于寡基因突变。

Congenital Hypothyroidism due to Oligogenic Mutations in Two Sudanese Families.

机构信息

1 Department of Medicine, University of Miami Miller School of Medicine, Miami, Florida.

2 Department of Paediatrics and Child Health, Faculty of Medicine, University of Khartoum, Khartoum, The Sudan.

出版信息

Thyroid. 2019 Feb;29(2):302-304. doi: 10.1089/thy.2018.0295. Epub 2018 Dec 18.

Abstract

Dyshormonogenic congenital hypothyroidism (CH) generally results from biallelic defects in thyroid hormone synthesis genes. Whole exome sequencing allows easier identification of multiple gene defects. Two Sudanese families with CH resulting from oligogenic defects identified by whole exome sequencing are presented. In family 1, the proposita with CH and goiter was heterozygous for three TPO, one TG, and one DUOX2 mutations, including three novel variants inherited from both parents. In family 2, two brothers with psychomotor delay and goiter were homozygous for digenic mutations in the DUOX2 and DUOX1 genes, while their asymptomatic parents were heterozygous. Accumulation of pathogenic mutations may contribute to CH.

摘要

先天性甲状腺功能减退症(CH)通常由甲状腺激素合成基因的双等位基因突变引起。外显子组测序可更轻松地鉴定多种基因缺陷。本文报道了两例苏丹 CH 家系,其致病原因为外显子组测序发现的寡基因缺陷。在 1 号家系中,患有 CH 和甲状腺肿的先证者为 TPO、TG 和 DUOX2 三个基因的杂合突变,包括从父母双方遗传的三个新变异。在 2 号家系中,两名患有精神运动发育迟缓伴甲状腺肿的兄弟为 DUOX2 和 DUOX1 基因的双等位基因突变纯合子,而无症状的父母为杂合子。致病性突变的积累可能导致 CH。

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