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染色体倒位作为体细胞重组表型的隐性疾病修饰因子。

Chromosomal inversions as a hidden disease-modifying factor for somatic recombination phenotypes.

机构信息

Department of Dermatology, Hokkaido University Graduate School of Medicine, Sapporo, Japan.

Department of Dermatology, Nagoya University Graduate School of Medicine, Nagoya, Japan.

出版信息

JCI Insight. 2018 Mar 22;3(6):97595. doi: 10.1172/jci.insight.97595.

Abstract

Heterozygous chromosomal inversions suppress recombination. Therefore, they may potentially influence recombination-associated phenotypes of human diseases, but no studies have verified this hypothesis. Here, we describe a 35-year-old man with severe congenital ichthyosis. Mutation analysis revealed a heterozygous splice-site mutation, c.1374-2A>G (p.Ser458Argfs*120), in KRT10 on 17q21.2. This mutation was previously reported in patients with ichthyosis with confetti type I (IWC-I), a prominent skin disease characterized by the frequent occurrence of recombination-induced reversion of pathogenic mutations. Intriguingly, the number of revertant skin areas in this patient is considerably reduced compared with typical IWC-I cases. G-banded karyotyping revealed that the patient harbors a heterozygous nonpathogenic inversion, inv(17)(p13q12), whose long-arm breakpoint was subsequently refined to chromosomal positions (chr17: 36,544,407-36,639,830) via FISH. Collectively, the only chance of revertant mosaicism through somatic recombination appears to involve recombination between the KRT10 mutation and the inversion breakpoint. Indeed, in the examined revertant spot, the KRT10 mutation was diminished by somatic recombination starting from chromosomal positions (chr17: 36,915,505-37,060,285) on 17q12. This study provides the first evidence to our knowledge implicating chromosomal inversions as a potential modifier of clinical phenotypes. Furthermore, the reduced occurrence of revertant spots in the recombination-suppressed patient suggests that somatic recombination is the main mechanism of revertant mosaicism in IWC-I.

摘要

杂合性染色体倒位抑制重组。因此,它们可能潜在地影响与人类疾病相关的重组表型,但尚未有研究验证这一假说。在这里,我们描述了一位 35 岁的严重先天性鱼鳞病患者。突变分析显示,17q21.2 上的 KRT10 存在杂合剪接位点突变,c.1374-2A>G(p.Ser458Argfs*120)。该突变之前在鱼鳞癣伴碎锦型 I(IWC-I)患者中报道过,这是一种以频繁发生致病突变重组回复为特征的突出皮肤病。有趣的是,与典型的 IWC-I 病例相比,该患者回复区皮肤的数量明显减少。G 带核型分析显示,该患者携带杂合性非致病性倒位,inv(17)(p13q12),其长臂断点随后通过 FISH 精确定位到染色体位置(chr17:36,544,407-36,639,830)。总的来说,通过体细胞重组产生回复型嵌合体的唯一机会似乎涉及 KRT10 突变和倒位断点之间的重组。事实上,在所研究的回复斑点中,KRT10 突变通过从染色体位置(chr17:36,915,505-37,060,285)开始的体细胞重组减少,在 17q12 上。本研究首次提供了染色体倒位作为临床表型潜在修饰因子的证据。此外,在重组抑制患者中回复斑点的发生率降低表明,体细胞重组是 IWC-I 中回复型嵌合体的主要机制。

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