对未受影响的家庭成员进行单基因运动神经元病/肌萎缩侧索硬化症的基因检测。

IF 3.7 2区 生物学 Q2 BIOCHEMISTRY & MOLECULAR BIOLOGY European Journal of Human Genetics Pub Date : 2024-11-05 DOI:10.1038/s41431-024-01718-4
Jade Howard, Amina Chaouch, Andrew G L Douglas, Rhona MacLeod, Jennifer Roggenbuck, Alisdair McNeill
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引用次数: 0

摘要

运动神经元病(MND)又称肌萎缩性脊髓侧索硬化症(ALS),在少数病例中是一种单基因疾病,为常染色体显性遗传。越来越多的 MND 患者要求进行基因检测,甚至接受基因诊断。因此,遗传咨询和预测性检测(即对未受影响的家庭成员进行检测)的请求预计同样会增加,同时还有症状前临床试验。尽管如此,目前尚无基于证据的 MND 预测性基因检测指南。本文概述了 MND 的基因组基础,特别关注 MND 最常见的单基因病因。然后,本文阐述了 MND 预测性检测的复杂性,包括不完全渗透性、临床和遗传异质性以及某些情况下的少基因致病机制等遗传特征。此外,有关MND预测性基因检测对社会心理影响的研究也很有限,研究表明,患者可能难以适应这一消息,部分原因是缺乏支持和随访。这突出表明,有必要为MND的预测性检测提供以证据为基础、针对特定疾病的指导。
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Genetic testing for monogenic forms of motor neuron disease/amyotrophic lateral sclerosis in unaffected family members.

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.

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来源期刊
European Journal of Human Genetics
European Journal of Human Genetics 生物-生化与分子生物学
CiteScore
9.90
自引率
5.80%
发文量
216
审稿时长
2 months
期刊介绍: The European Journal of Human Genetics is the official journal of the European Society of Human Genetics, publishing high-quality, original research papers, short reports and reviews in the rapidly expanding field of human genetics and genomics. It covers molecular, clinical and cytogenetics, interfacing between advanced biomedical research and the clinician, and bridging the great diversity of facilities, resources and viewpoints in the genetics community. Key areas include: -Monogenic and multifactorial disorders -Development and malformation -Hereditary cancer -Medical Genomics -Gene mapping and functional studies -Genotype-phenotype correlations -Genetic variation and genome diversity -Statistical and computational genetics -Bioinformatics -Advances in diagnostics -Therapy and prevention -Animal models -Genetic services -Community genetics
期刊最新文献
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