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FBXO11 变异与中国受影响个体的智力残疾和可变临床表现有关。

FBXO11 variants are associated with intellectual disability and variable clinical manifestation in Chinese affected individuals.

机构信息

Department of Gynecology and Obstetrics, The Second Affiliated Hospital of Chongqing Medical University, Chongqing, 400000, China.

Center for Clinical Genetics and Genomics, DIAN Diagnostics, Hangzhou, 310058, Zhejiang, China.

出版信息

J Hum Genet. 2024 Aug;69(8):391-400. doi: 10.1038/s10038-024-01255-4. Epub 2024 May 13.

Abstract

F-box protein 11 (FBXO11) is a member of F-Box protein family, which has recently been proved to be associated with intellectual developmental disorder with dysmorphic facies and behavioral abnormalities (IDDFBA, OMIM: 618089). In this study, 12 intellectual disability individuals from 5 Chinese ID families were collected, and whole exome sequencing (WES), sanger sequencing, and RNA sequencing (RNA-seq) were conducted. Almost all the affected individuals presented with mild to severe intellectual disability (12/12), global developmental delay (10/12), speech and language development delay (8/12) associated with a range of alternate features including increased body weight (7/12), short stature (6/12), seizures (3/12), reduced visual acuity (4/12), hypotonia (1/12), and auditory hallucinations and hallucinations (1/12). Distinguishingly, malformation was not observed in all the affected individuals. WES analysis showed 5 novel FBXO11 variants, which include an inframe deletion variant, a missense variant, two frameshift variants, and a partial deletion of FBXO11 (exon 22-23). RNA-seq indicated that exon 22-23 deletion of FBXO11 results in a new mRNA structure. Conservation and protein structure prediction demonstrated deleterious effect of these variants. The DEGs analysis revealed 148 differentially expressed genes shared among 6 affected individuals, which were mainly associated with genes of muscle and immune system. Our research is the first report of FBXO11-associated IDDFBA in Chinese individuals, which expands the genetic and clinical spectrum of this newly identified NDD/ID syndrome.

摘要

F-box 蛋白 11(FBXO11)是 F-Box 蛋白家族的一员,最近已被证实与伴有畸形面容和行为异常的智力发育障碍(IDDFBA,OMIM:618089)有关。在这项研究中,收集了来自 5 个中国 ID 家系的 12 名智力障碍个体,进行了全外显子组测序(WES)、Sanger 测序和 RNA 测序(RNA-seq)。几乎所有受影响的个体都表现出轻度至重度智力障碍(12/12)、全面发育迟缓(10/12)、言语和语言发育迟缓(8/12),并伴有一系列其他特征,包括体重增加(7/12)、身材矮小(6/12)、癫痫发作(3/12)、视力下降(4/12)、张力减退(1/12)和听觉幻觉和幻觉(1/12)。值得注意的是,所有受影响的个体均未观察到畸形。WES 分析显示 5 种新型 FBXO11 变异体,包括移码缺失变异体、错义变异体、2 种移码变异体和 FBXO11 的部分缺失(exon22-23)。RNA-seq 表明 FBXO11 的 exon22-23 缺失导致新的 mRNA 结构。保守性和蛋白质结构预测表明这些变异体具有有害影响。DEGs 分析显示,在 6 名受影响的个体中共有 148 个差异表达基因,这些基因主要与肌肉和免疫系统的基因有关。我们的研究是中国人群中首次报道的 FBXO11 相关 IDDFBA,扩展了这一新确定的 NDD/ID 综合征的遗传和临床谱。

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