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一个埃及人家族患有 H 综合征,其病因是 SLC29A3 中的一个新突变,该病症与色素性多毛性皮肤异色症伴胰岛素依赖型糖尿病和 Faisalabad 组织细胞增多症具有重叠特征。

An Egyptian family with H syndrome due to a novel mutation in SLC29A3 illustrating overlapping features with pigmented hypertrichotic dermatosis with insulin-dependent diabetes and Faisalabad histiocytosis.

机构信息

Department of Pediatrics, Faculty of Medicine, Ain Shams University, Cairo, Egypt.

出版信息

Pediatr Diabetes. 2013 Sep;14(6):466-72. doi: 10.1111/j.1399-5448.2012.00925.x. Epub 2012 Sep 18.

Abstract

The SLC29A3 gene, encoding hENT3, a member of the equilibrative nucleoside transporter family, has recently been found mutated in Faisalabad histiocytosis, pigmented hypertrichotic dermatosis with insulin-dependent diabetes, familial sinus histiocytosis with massive lymphadenopathy (SHML), and H syndromes. We here report clinical and genetic findings of an Egyptian family with H syndrome. We describe two siblings, a 19-yr old girl and a 15-yr old boy, of consanguineous parents. From 5 yr of age, the girl developed bilateral flexion deformity of interphalengeal joints and insulin-dependent diabetes mellitus. At age 7 yr, prominent hyperpigmented patches appeared on the skin at lower limbs, genitalia, and trunk. On clinical examination, she had hepatosplenomegaly, generalized lymphadenopathy, left ventricular hypertrophy, sensorineural hearing loss, hypogonadism, short stature, and characteristic dysmorphic features. Her brother had fixed flexion contractures of the feet, profound sensorineural hearing loss, characteristic dysmorphic features, but no diabetes. DNA sequence analysis revealed a homozygous mutation (c.300+1G>C) in SLC29A3 in both siblings. The phenotype and genotype of the siblings were compatible with that of the H syndrome, although the features were overlapping with those found in pigmented hypertrichotic dermatosis with insulin-dependent diabetes, familial SHML, and Faisalabad histiocytosis, indicating that these four syndromes may be regarded as one disease with varying phenotypic features. A new, common name for these conditions is warranted.

摘要

SLC29A3 基因,编码 hENT3,是平衡核苷转运体家族的成员,最近在 Faisalabad 组织细胞增生症、色素性多毛性皮肤病伴胰岛素依赖型糖尿病、家族性鼻窦组织细胞增生症伴广泛淋巴结病(SHML)和 H 综合征中发现突变。我们在此报告一例埃及 H 综合征家系的临床和遗传学发现。我们描述了一对 19 岁的女孩和 15 岁的男孩,他们的父母是近亲。从 5 岁开始,女孩出现双侧指间关节弯曲畸形和胰岛素依赖型糖尿病。7 岁时,下肢、生殖器和躯干出现明显的色素沉着斑块。体格检查时,她有肝脾肿大、全身淋巴结肿大、左心室肥厚、感觉神经性听力损失、性腺功能减退、身材矮小和特征性的畸形特征。她的哥哥有足固定性弯曲挛缩、严重的感觉神经性听力损失、特征性的畸形特征,但没有糖尿病。DNA 序列分析显示,在这对兄妹中 SLC29A3 存在纯合突变(c.300+1G>C)。兄妹俩的表型和基因型与 H 综合征相符,尽管其特征与色素性多毛性皮肤病伴胰岛素依赖型糖尿病、家族性 SHML 和 Faisalabad 组织细胞增生症重叠,表明这四种综合征可能被视为一种具有不同表型特征的疾病。这些疾病需要一个新的、通用的名称。

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