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沙特阿拉伯一位埃勒斯-当洛斯综合征患者出现罕见神经系统表现和 TNXB 基因新的纯合变异。

Rare neurological manifestations in a Saudi Arabian patient with Ehlers-Danlos syndrome and a novel homozygous variant in the TNXB gene.

机构信息

Neurology Department, Neuroscience Centre, King Fahad Specialist Hospital-Dammam, Dammam, Saudi Arabia.

Genetic Unit, Pathology and Laboratory Medicine Department, King Fahad Specialist Hospital-Dammam, Dammam, Saudi Arabia.

出版信息

Am J Med Genet A. 2022 Feb;188(2):618-623. doi: 10.1002/ajmg.a.62539. Epub 2021 Oct 11.

Abstract

We report a 38-year-old Saudi male with Ehlers-Danlos Syndrome (EDS). The patient presented with rare and unusual neurological manifestations, including but not limited to ophthalmoplegia and myopathic pattern on his electromyography. In addition to hand weakness, there was skin hyperextensibility, joint hyperflexibility, and frontal baldness. Next-generation sequencing was performed on target exon sequences, using whole exome sequencing and Burrows-Wheeler Aligner for alignment/base calling. Genome Analysis Toolkit and reference genome Homo sapiens (UCSC hg19) were used for sequence processing and analysis. Variant classification was done according to standard international recommendations. A novel homozygous variant, NM_019105.6: c.8488C>T p.(Gln2830*), was detected in the TNXB gene. This variant is not reported in the literature nor dbSNP or gnomAD databases. Additionally, this variant is predicted to create a premature stop codon and produce a truncated protein or nonsense-mediated mRNA decay. Hence, it is classified as a likely pathogenic variant. The same point variant was found in a heterozygous state in the patient's father and sister. Both presented with milder symptoms associated with Ehlers-Danlos syndromes and heritable connective tissue disorders. Therefore, the patient was diagnosed as a tenascin-X (TNX) deficient type of EDS known as classical-like Ehlers-Danlos syndrome. TNX deficient patients may present with clinical and electrophysiological manifestations that are unusual in EDS like frontal baldness, ophthalmoplegia, and myotonia, which mimic myotonic dystrophy type I. Clinicians should be aware of the potential overlap of symptoms among these two diseases to ensure correct diagnosis is made.

摘要

我们报告了一例 38 岁的沙特男性患有埃勒斯-当洛斯综合征(EDS)。该患者表现出罕见且不寻常的神经表现,包括但不限于眼肌麻痹和肌电图上的肌病模式。除了手部无力外,还有皮肤过度伸展、关节过度伸展和额部秃顶。对靶向外显子序列进行了下一代测序,使用全外显子组测序和 Burrows-Wheeler Aligner 进行比对/碱基调用。基因组分析工具包和参考基因组 Homo sapiens(UCSC hg19)用于序列处理和分析。根据标准国际建议对变体进行分类。在 TNXB 基因中检测到一个新的纯合变体 NM_019105.6:c.8488C>T p.(Gln2830*)。该变体在文献中、dbSNP 或 gnomAD 数据库中均未报道。此外,该变体预计会产生一个提前终止密码子,并产生截短蛋白或无义介导的 mRNA 降解。因此,它被归类为可能的致病变体。该相同的点变体在患者的父亲和姐姐中以杂合状态发现。两者均表现出与埃勒斯-当洛斯综合征和遗传性结缔组织疾病相关的较轻症状。因此,该患者被诊断为经典型埃勒斯-当洛斯综合征,即 tenascin-X(TNX)缺乏型 EDS。TNX 缺乏患者可能表现出不寻常的 EDS 临床和电生理表现,如额部秃顶、眼肌麻痹和肌强直,类似于 I 型肌强直性营养不良。临床医生应注意这两种疾病之间症状的潜在重叠,以确保正确诊断。

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