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全外显子测序和多基因评估瑞典严重发育性语言障碍队列。

Whole exome sequencing and polygenic assessment of a Swedish cohort with severe developmental language disorder.

机构信息

Center of Neurodevelopmental Disorders (KIND), Centre for Psychiatry Research, Department of Women's and Children's Health, Karolinska Institutet, Region Stockholm, Stockholm, Sweden.

Astrid Lindgren Children's Hospital, Karolinska University Hospital, Region Stockholm, Stockholm, Sweden.

出版信息

Hum Genet. 2024 Feb;143(2):169-183. doi: 10.1007/s00439-023-02636-z. Epub 2024 Feb 1.

Abstract

Developmental language disorder (DLD) overlaps clinically, genetically, and pathologically with other neurodevelopmental disorders (NDD), corroborating the concept of the NDD continuum. There is a lack of studies to understand the whole genetic spectrum in individuals with DLD. Previously, we recruited 61 probands with severe DLD from 59 families and examined 59 of them and their families using microarray genotyping with a 6.8% diagnostic yield. Herein, we investigated 53 of those probands using whole exome sequencing (WES). Additionally, we used polygenic risk scores (PRS) to understand the within family enrichment of neurodevelopmental difficulties and examine the associations between the results of language-related tests in the probands and language-related PRS. We identified clinically significant variants in four probands, resulting in a 7.5% (4/53) molecular diagnostic yield. Those variants were in PAK2, MED13, PLCB4, and TNRC6B. We also prioritized additional variants for future studies for their role in DLD, including high-impact variants in PARD3 and DIP2C. PRS did not explain the aggregation of neurodevelopmental difficulties in these families. We did not detect significant associations between the language-related tests and language-related PRS. Our results support using WES as the first-tier genetic test for DLD as it can identify monogenic DLD forms. Large-scale sequencing studies for DLD are needed to identify new genes and investigate the polygenic contribution to the condition.

摘要

发育性语言障碍(DLD)在临床上、遗传上和病理学上与其他神经发育障碍(NDD)重叠,证实了 NDD 连续体的概念。目前缺乏研究来了解 DLD 个体的整个遗传谱。先前,我们从 59 个家庭中招募了 61 名严重 DLD 的先证者,并对其中的 59 名先证者及其家属进行了微阵列基因分型检测,诊断率为 6.8%。在此,我们使用全外显子组测序(WES)对其中的 53 名先证者进行了研究。此外,我们还使用多基因风险评分(PRS)来了解神经发育困难在家庭内的富集情况,并检查先证者语言相关测试结果与语言相关 PRS 之间的关联。我们在 4 名先证者中发现了具有临床意义的变异,分子诊断率为 7.5%(4/53)。这些变异位于 PAK2、MED13、PLCB4 和 TNRC6B 基因中。我们还为进一步研究这些基因在 DLD 中的作用,确定了其他变异作为优先研究对象,包括 PARD3 和 DIP2C 基因中的高影响变异。PRS 并不能解释这些家庭中神经发育困难的聚集。我们没有检测到语言相关测试与语言相关 PRS 之间存在显著关联。我们的研究结果支持将 WES 作为 DLD 的一线基因检测方法,因为它可以识别单基因 DLD 形式。需要对 DLD 进行大规模测序研究,以确定新的基因,并研究该疾病的多基因贡献。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/8caf/10881898/12f5039ca758/439_2023_2636_Fig1_HTML.jpg

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