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患有CHD3致病变体个体的神经行为特征

Neurobehavioral profile of individuals with pathogenic variants in CHD3.

作者信息

Ionescu Anca, Mazur-Lainé Emmanuelle, Proteau-Lemieux Mélodie, Knoth Inga S, Vachon Keely, Whitlock Kerri, Biag Hazel Maridith Barlanhan, Rabouhi Nazim, Van Heesbeen Hendrikus J, Jacquemont Sébastien, Hessl David, Abbeduto Leonard, Anagnostou Evdokia, Bolduc François, Hagerman Randi J, Campeau Philippe M, Lippé Sarah

机构信息

University of Montreal, Montreal, QC, Canada.

Azrieli Research Center of the Sainte-Justine University Hospital, Montreal, QC, Canada.

出版信息

Eur J Hum Genet. 2025 Aug 19. doi: 10.1038/s41431-025-01926-6.

Abstract

Snijders Blok-Campeau syndrome (SNIBCPS), a neurodevelopmental disorder first described in 2018, is caused by heterozygous pathogenic variants in CHD3. Its encoded protein plays a crucial role in the development of the nervous system of embryos. While phenotypic traits have been broadly defined, i.e., global neurodevelopmental delays such as intellectual disabilities and delayed speech acquisition, and physical features such as characteristic facial features and macrocephaly, the phenotypic spectrum has not been further assessed. We present the neurobehavioral profile of 38 individuals with variants in CHD3 and compare it to the ones of autism spectrum disorder (ASD) and Fragile X syndrome (FXS) cohorts. Profound clinical deficits were found in adaptive functioning, communication skills, and sensorimotor functioning in most SNIBCPS participants. Similarities between FXS and SNIBCPS cohorts were unveiled, characterized by diminished levels of global adaptive behavior and adaptive functioning in the social and communication domains. Nevertheless, despite profound challenges in global adaptive behavior in SNIBCPS, we reveal the social domain as showing the highest adaptive levels alongside minimal emotional/behavioral issues within the sample, suggesting relative strengths inherent to SNIBCPS. This study enriches the scarce SNIBCPS literature by delineating the neurobehavioral phenotypic spectrum of SNIBCPS and by innovating comparisons with clinically akin neurodevelopmental disorders.

摘要

斯奈德氏布洛克-坎波综合征(SNIBCPS)是一种于2018年首次被描述的神经发育障碍,由CHD3基因的杂合致病变异引起。其编码的蛋白质在胚胎神经系统发育中起关键作用。虽然已大致定义了表型特征,即智力残疾和语言习得延迟等全面的神经发育迟缓,以及特征性面部特征和巨头症等身体特征,但尚未进一步评估表型谱。我们展示了38名CHD3基因变异个体的神经行为概况,并将其与自闭症谱系障碍(ASD)和脆性X综合征(FXS)队列的概况进行比较。在大多数SNIBCPS参与者中,发现其在适应性功能、沟通技巧和感觉运动功能方面存在严重临床缺陷。揭示了FXS和SNIBCPS队列之间的相似之处,其特征是整体适应性行为水平降低以及社交和沟通领域的适应性功能受损。然而,尽管SNIBCPS在整体适应性行为方面面临巨大挑战,但我们发现社交领域在样本中显示出最高适应性水平,同时情绪/行为问题最少,这表明SNIBCPS具有相对优势。本研究通过描绘SNIBCPS的神经行为表型谱以及创新与临床类似神经发育障碍的比较,丰富了稀缺的SNIBCPS文献。

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