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WNK1 基因中的一种新的致病性突变导致三兄妹中的 HSAN 型 II。

A Novel Pathogenic Mutation in WNK1 Gene Causing HSAN Type II in Three Siblings.

机构信息

Department of Medical Genetics, Faculty of Medicine, Tabriz University of Medical Sciences, Tabriz, Iran.

出版信息

J Mol Neurosci. 2024 Oct 23;74(4):99. doi: 10.1007/s12031-024-02282-8.

Abstract

Hereditary sensory and autonomic neuropathy (HSAN) is a rare genetic disorder that primarily affects the peripheral nervous system, leading to a progressive loss of the ability to perceive pain, temperature, and touch. This condition can result in severe complications, including injuries and infections due to the inability to feel pain. HSAN is classified into nine types, with types I and VII exhibiting autosomal dominant inheritance, while the others follow an autosomal recessive pattern. In this study, we examined three affected brothers of Turkish Azeri descent, aged 20, 23, and 25 years. They presented symptoms such as a lack of temperature and pain sensation, frequent wounds and infections, self-harm, and hyperkeratosis. To identify the genetic cause of their condition, whole-exome sequencing (WES) was performed, followed by Sanger sequencing to confirm the findings. The results revealed a homozygous likely pathogenic nonsense mutation, c.2971C > T (p.Arg991Ter), in exon 9 of the WNK1 gene. This mutation results in the truncation of three isoforms of the WNK1 protein, which are essential for pain perception. This discovery enhances our understanding of HSAN and highlights the importance of genetic testing for accurate diagnosis and future screening.

摘要

遗传性感觉运动神经病(HSAN)是一种罕见的遗传性疾病,主要影响外周神经系统,导致感知疼痛、温度和触觉的能力逐渐丧失。这种情况可能会导致严重的并发症,包括因无法感知疼痛而导致的受伤和感染。HSAN 分为九种类型,I 型和 VII 型表现为常染色体显性遗传,而其他类型则遵循常染色体隐性遗传模式。在这项研究中,我们检查了三个受影响的土耳其阿塞拜疆裔兄弟,年龄分别为 20 岁、23 岁和 25 岁。他们表现出缺乏温度和疼痛感觉、频繁的伤口和感染、自残和过度角化等症状。为了确定他们疾病的遗传原因,我们进行了全外显子组测序(WES),然后进行 Sanger 测序以确认发现。结果显示,WNK1 基因外显子 9 中的 c.2971C>T(p.Arg991Ter)纯合可能致病无义突变。该突变导致 WNK1 蛋白的三个同工型截断,这对于疼痛感知至关重要。这一发现加深了我们对 HSAN 的认识,并强调了遗传测试对于准确诊断和未来筛查的重要性。

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