致病性KCNH2变异在言语迟缓和低风险2型长QT综合征的同卵双胞胎中。

IF 1.2 4区 医学 Q3 CLINICAL NEUROLOGY
Katia Margiotti, Marco Fabiani, Costanza Zangheri, Antonella Cima, Francesca Monaco, Francesco Libotte, Chiara Ali', Maria Antonietta Barone, Antonella Viola, Alvaro Mesoraca, Claudio Giorlandino
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引用次数: 0

摘要

2型长QT综合征(2型LQTS)是一种由KCNH2基因致病性变异引起的心脏通道病变,通常与心脏复极延迟和心律失常风险增加有关。虽然传统上认为其影响是心脏,但新兴研究表明KCNH2功能障碍在神经系统疾病中的潜在作用。我们描述了携带致病移码变异KCNH2 c.2959_2960delCT (p.Leu987Valfs*131;Rs748706373),遗传自他们无症状的父亲。在临床上,这对双胞胎都表现出严重的语言迟缓、缺乏指指、社交障碍以及与神经障碍一致的刻板行为特征。诊断诊断检测包括全外显子组测序(WES)、染色体微阵列(aCGH)和脆性X染色体筛查。KCNH2变异是唯一具有临床意义的发现。通过心电图和24小时动态心电图监测的心脏评估显示,双胞胎及其父亲均未出现明显的QT延长或心律失常发作。无晕厥、癫痫发作或心脏事件史。该报告支持KCNH2变异在2型LQTS中的可变表达性和不完全外显性,并提出KCNH2功能障碍可能有助于神经表型表现的可能性。KCNH2与神经系统疾病之间的因果关系尚待确定。尽管该家系的全基因组测序仍有待完成,但KCNH2与神经系统疾病之间的潜在关联因其独特的单合子表现和没有已知的围产期并发症而得到加强。需要进一步的研究来阐明KCNH2变异及其对神经系统疾病的贡献之间的关联,无论是通过直接的神经效应还是通过未被识别的围产期心律失常事件间接的。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Pathogenic KCNH2 variant in monozygotic twins with speech delay and lower risk type 2 long QT syndrome.

Type 2 Long QT Syndrome (type 2 LQTS) is a cardiac channelopathy caused by pathogenic variants in the KCNH2 gene, often associated with delayed cardiac repolarization and increased risk of arrhythmias. While its impact is traditionally considered cardiac, emerging studies suggest a potential role of KCNH2 dysfunction in neurogical disorders. We describe monozygotic twin sisters carrying the pathogenic frameshift variant KCNH2 c.2959_2960delCT (p.Leu987Valfs*131; rs748706373), inherited from their asymptomatic father. Clinically, both twins presented with severe language delay, absence of pointing, impaired social interaction, and stereotyped behaviors features consistent with neurogical disorders. Diagnostic diagnostic testing included whole exome sequencing (WES), chromosomal microarray (aCGH), and Fragile X screening. The KCNH2 variant emerged as the sole clinically significant finding. Cardiac evaluation through ECG and 24-hour Holter monitoring revealed no significant QT prolongation or arrhythmic episodes in either the twins or their father. No history of syncope, seizures, or cardiac events was reported. This report supports the variable expressivity and incomplete penetrance of KCNH2 variants in type 2 LQTS and raises the possibility that KCNH2 dysfunction may contribute to neurogical phenotypes manifestations. Causality remains to be established between KCNH2 and neurologic disorders. Though whole-genome sequencing remains to be completed in this pedigree, the potential association between KCNH2 and neurologic disorders is strengthened by the unique monozygotic presentation and the absence of known perinatal complications. Further studies are needed to clarify the association between KCNH2 variants and their contribution to neurological disorders, either through direct neural effects or indirectly via unrecognized perinatal arrhythmic events.

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来源期刊
Neurogenetics
Neurogenetics 医学-临床神经学
CiteScore
3.90
自引率
0.00%
发文量
24
审稿时长
6 months
期刊介绍: Neurogenetics publishes findings that contribute to a better understanding of the genetic basis of normal and abnormal function of the nervous system. Neurogenetic disorders are the main focus of the journal. Neurogenetics therefore includes findings in humans and other organisms that help understand neurological disease mechanisms and publishes papers from many different fields such as biophysics, cell biology, human genetics, neuroanatomy, neurochemistry, neurology, neuropathology, neurosurgery and psychiatry. All papers submitted to Neurogenetics should be of sufficient immediate importance to justify urgent publication. They should present new scientific results. Data merely confirming previously published findings are not acceptable.
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