病例报告:第一例由确认的马赛克无症状携带者遗传而引起的终末性肌原纤维肌病。

IF 2.8 3区 生物学 Q2 GENETICS & HEREDITY
Frontiers in Genetics Pub Date : 2025-06-18 eCollection Date: 2025-01-01 DOI:10.3389/fgene.2025.1597851
Jelle Vlaeminck, Sophie Uyttebroeck, Elke De Schutter, Ann Cordenier, Shauni Wellekens, Erwin Ströker, Kelly De Rooms, Christine Helsen, Frederik J Hes, Philippe Giron
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引用次数: 0

摘要

desmin相关的肌原纤维肌病是一种由DES基因(MIM*125660)的致病性变异引起的遗传性疾病,改变desmin,一种对肌节完整性至关重要的肌肉特异性中间纤维。这种情况表现为骨骼肌病、心肌病和传导异常。对指数患者及其家庭成员的遗传咨询因表达性变化、不完全外显和新发而变得复杂。无症状父母的嵌合现象可以模糊遗传模式,特别是当血液中低等级的嵌合变异可能被遗漏时。在DES的情况下,马赛克载体以前没有被描述过。我们描述了一例24岁女性因杂合致病性NM_001927.4 (DES):c而被诊断为肾小球相关性肌病。1216C>T, p.a g406trp变体。对其无症状父母进行靶向Sanger测序的级联检测表明,母亲是致病变异的马赛克载体,这一点通过下一代测序得到了证实。先证者的兄弟姐妹没有携带DES c.1216C>T变异。我们报告了第一例无症状个体中致病性DES变异的马赛克携带病例,并随后由后代遗传,导致与desin相关的肌病。本报告强调了级联检测在遗传性疾病中的重要性,重点是嵌合体,即使该指数的亲生父母没有症状,并且怀疑从头出现。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Case Report: A first case of desmin-related myofibrillar myopathy due to inheritance from a confirmed mosaic asymptomatic carrier.

Desmin-related myofibrillar myopathy is a hereditary disorder caused by pathogenic variants in the DES gene (MIM*125660), altering desmin, a muscle-specific intermediate filament which is crucial for sarcomere integrity. This condition presents with skeletal myopathy, cardiomyopathy, and conduction abnormalities. Genetic counselling for index patients and their family members is complicated by variable expressivity, incomplete penetrance, and de novo occurrence. Mosaicism in asymptomatic parents can obscure inheritance patterns, particularly when low-grade mosaic variants in blood may be missed. In case of DES, mosaic carriership has not been described before. We describe a case of a 24-year-old female diagnosed with desmin-related myopathy due to a heterozygous pathogenic NM_001927.4 (DES):c.1216C>T, p.Arg406Trp variant. Cascade testing using targeted Sanger sequencing of her asymptomatic parents suggested the mother is a mosaic carrier of the pathogenic variant, which was confirmed though next-generation sequencing. The proband's siblings did not carry the DES c.1216C>T variant. We report the first documented case of mosaic carriership of a pathogenic DES variant in an asymptomatic individual and subsequent inheritance by the offspring, leading to desmin-related myopathy. This report highlights the importance of cascade testing in hereditary disorders with a focus on mosaicism, even when the index's biological parents are asymptomatic, and de novo emergence is suspected.

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来源期刊
Frontiers in Genetics
Frontiers in Genetics Biochemistry, Genetics and Molecular Biology-Molecular Medicine
CiteScore
5.50
自引率
8.10%
发文量
3491
审稿时长
14 weeks
期刊介绍: Frontiers in Genetics publishes rigorously peer-reviewed research on genes and genomes relating to all the domains of life, from humans to plants to livestock and other model organisms. Led by an outstanding Editorial Board of the world’s leading experts, this multidisciplinary, open-access journal is at the forefront of communicating cutting-edge research to researchers, academics, clinicians, policy makers and the public. The study of inheritance and the impact of the genome on various biological processes is well documented. However, the majority of discoveries are still to come. A new era is seeing major developments in the function and variability of the genome, the use of genetic and genomic tools and the analysis of the genetic basis of various biological phenomena.
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