在家族性 GRIN2A 相关疾病中,双配子体嵌合是一种不寻常的遗传机制。

IF 3.5 2区 医学 Q2 GENETICS & HEREDITY Journal of Medical Genetics Pub Date : 2024-09-24 DOI:10.1136/jmg-2024-110101
Valentina Cetica, Mara Cavallin, Maria Luisa Ricci, Claudia Mandorlini, Emanuele Bartolini, Elena Parrini, Renzo Guerrini
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引用次数: 0

摘要

一位母亲将同一核苷酸上的两种不同致病变体遗传给了她的两个患儿,我们旨在描述这位母亲的 GRIN2A 基因中的双染色体嵌合现象。我们研究了一名患有癫痫和智力障碍的男孩,以及他的姐姐和母亲,他们都有语言障碍和学习困难,但没有癫痫。我们在这名男孩身上发现了从其母亲那里遗传的 GRIN2A(c.1008-1G>A)剪接位点变异。随后对他的妹妹进行的检测发现,在同一核苷酸c.1008-1G>T上存在不同的变异,该变异也存在于母亲的DNA中,等位基因频率为3.9%。在同一核苷酸上同时出现两个突变事件是极其罕见的。由于偶然发生的可能性不大,我们假设碱基错配可能会带来不稳定性,从而引发第二次突变。在这个家庭中,母亲携带三个等位基因,其中一个频率非常低。这种复杂的遗传情况给诊断带来了挑战,因为低水平的镶嵌可能无法通过传统方法检测出来。应用特定的技术变得至关重要,因为双重嵌合可能比预期的更为普遍,严重影响诊断的准确性和遗传咨询。
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Double gonosomal mosaicism as an unusual hereditary mechanism in familial GRIN2A-related disorder.

We aim to describe double gonosomal mosaicism in the GRIN2A gene in a mother who passed on two different pathogenic variants at the same nucleotide to her two affected children. We studied a boy with epilepsy and intellectual disability, along with his sister and mother who exhibited language impairment and learning difficulties without epilepsy. We identified in the proband a splice-site variant in GRIN2A (c.1008-1G>A) inherited from his mother. Subsequent testing of his sister revealed a different change at the same nucleotide c.1008-1G>T, which was also present in the mother's DNA at 3.9% allele frequency. The co-occurrence of two mutational events at the same nucleotide is extremely rare. Since a chance occurrence is unlikely, we hypothesise that a base mismatch may introduce instability triggering a second event. In this family, the mother carries three alleles, of which one is at very low frequency. This complex genetic landscape poses diagnostic challenges since low-level mosaicism may escape detection via conventional methods. Applying specific technology becomes crucial, as double mosaicism might prove to be more prevalent than anticipated severely impacting diagnostic accuracy and genetic counselling.

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来源期刊
Journal of Medical Genetics
Journal of Medical Genetics 医学-遗传学
CiteScore
7.60
自引率
2.50%
发文量
92
审稿时长
4-8 weeks
期刊介绍: Journal of Medical Genetics is a leading international peer-reviewed journal covering original research in human genetics, including reviews of and opinion on the latest developments. Articles cover the molecular basis of human disease including germline cancer genetics, clinical manifestations of genetic disorders, applications of molecular genetics to medical practice and the systematic evaluation of such applications worldwide.
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