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对未患病家庭成员进行单基因形式运动神经元病/肌萎缩侧索硬化症的基因检测。

Genetic testing for monogenic forms of motor neuron disease/amyotrophic lateral sclerosis in unaffected family members.

作者信息

Howard Jade, Chaouch Amina, Douglas Andrew G L, MacLeod Rhona, Roggenbuck Jennifer, McNeill Alisdair

机构信息

Division of Neuroscience and Neuroscience Institute, The University of Sheffield, Sheffield, UK.

Manchester Centre for Clinical Neuroscience, Salford Royal NHS Foundation Trust, Northern Care Alliance, Stott Lane, M6 8HD, UK.

出版信息

Eur J Hum Genet. 2025 Jan;33(1):7-13. doi: 10.1038/s41431-024-01718-4. Epub 2024 Nov 5.

Abstract

Motor neuron disease (MND), also referred to as amyotrophic lateral sclerosis (ALS), is a monogenic disease in a minority of cases, with autosomal dominant inheritance. Increasing numbers of people with MND are requesting genetic testing, and indeed receiving a genetic diagnosis. Consequently, requests for genetic counselling and predictive testing (i.e. of unaffected family members) are similarly expected to rise, alongside pre-symptomatic clinical trials. Despite this, there is no evidence-based guideline for predictive genetic testing in MND. This paper provides an overview of the genomic basis of MND, focusing specifically on the most common monogenic causes of MND. It then lays out the complexities of MND predictive testing, including the genetic landscape characterised by incomplete penetrance, clinical and genetic heterogeneity, and an oligogenic mechanism of pathogenesis in some cases. Additionally, there is limited research on the psychosocial impact of predictive genetic testing for MND, with studies suggesting potential difficulty in adjusting to the news, in part due to a lack of support and follow-up. This underscores a case for evidence-based, disease-specific guidance for predictive testing in MND.

摘要

运动神经元病(MND),也被称为肌萎缩侧索硬化症(ALS),在少数情况下是一种单基因疾病,具有常染色体显性遗传特征。越来越多的运动神经元病患者要求进行基因检测,并且确实获得了基因诊断结果。因此,与症状前临床试验一样,对遗传咨询和预测性检测(即对未患病家庭成员的检测)的需求预计也会增加。尽管如此,目前尚无关于运动神经元病预测性基因检测的循证指南。本文概述了运动神经元病的基因组基础,特别关注运动神经元病最常见的单基因病因。接着阐述了运动神经元病预测性检测的复杂性,包括以不完全外显率、临床和遗传异质性以及某些情况下的寡基因致病机制为特征的遗传格局。此外,关于运动神经元病预测性基因检测对心理社会影响的研究有限,研究表明,部分由于缺乏支持和后续跟进,患者在适应检测结果方面可能存在困难。这凸显了为运动神经元病预测性检测制定循证的、针对该疾病的指导意见的必要性。

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