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肌肉组织相关基因中的序列变异可能决定脑瘫患者肌肉挛缩的严重程度。

Sequence variants in muscle tissue-related genes may determine the severity of muscle contractures in cerebral palsy.

机构信息

Department of Neuroscience and Pharmacology, Faculty of Health and Medical Sciences, University of Copenhagen, Copenhagen, Denmark.

Department of Forensic Medicine, Section of Forensic Genetics, Faculty of Health and Medical Sciences, University of Copenhagen, Copenhagen, Denmark.

出版信息

Am J Med Genet B Neuropsychiatr Genet. 2019 Jan;180(1):12-24. doi: 10.1002/ajmg.b.32693. Epub 2018 Nov 23.

Abstract

Muscle contractures are a common complication to cerebral palsy (CP). The purpose of this study was to evaluate whether individuals with CP carry specific gene variants of important structural genes that might explain the severity of muscle contractures. Next-generation-sequencing (NGS) of 96 candidate genes associated with muscle structure and metabolism were analyzed in 43 individuals with CP (Gross Motor Function classification system [GMFCS] I, n=10; GMFCS II, n=14; GMFCS III, n=19) and four control participants. In silico analysis of the identified variants was performed. The variants were classified into four categories ranging from likely benign (VUS0) to highly likely functional effect (VUS3). All individuals with CP were classified and grouped according to their GMFCS level: Statistical comparisons were made between GMFCS groups. Kruskal-Wallis tests showed significantly more VUS2 variants in the genes COL4 (GMFCS I-III; 1, 1, 5, respectively [p < .04]), COL5 (GMFCS I-III; 1, 1, 5 [p < .04]), COL6 (GMFCS I-III; 0, 4, 7 [p < .003]), and COL9 (GMFCS I-III; 1, 1, 5 [p < .04]), in individuals with CP within GMFCS Level III when compared to the other GMFCS levels. Furthermore, significantly more VUS3 variants in COL6 (GMFCS I-III; 0, 5, 2 [p < .01]) and COL7 (GMFCS I-III; 0, 3, 0 [p < .04]) were identified in the GMFCS II level when compared to the other GMFCS levels. The present results highlight several candidate gene variants in different collagen types with likely functional effects in individuals with CP.

摘要

肌肉挛缩是脑瘫(CP)的常见并发症。本研究旨在评估 CP 患者是否携带可能解释肌肉挛缩严重程度的重要结构基因的特定基因变异。对 43 名 CP 患者(GMFCS 分级系统 I 级,n=10;GMFCS 分级系统 II 级,n=14;GMFCS 分级系统 III 级,n=19)和 4 名对照参与者的 96 个候选基因与肌肉结构和代谢相关的基因进行了下一代测序(NGS)分析。对鉴定出的变异进行了计算机分析。将这些变异分为四个类别,从可能良性(VUS0)到高度可能有功能效应(VUS3)。所有 CP 患者均根据 GMFCS 水平进行分类和分组:对 GMFCS 组之间进行了统计比较。Kruskal-Wallis 检验显示,COL4 基因(GMFCS I-III;分别为 1、1、5,p<0.04)、COL5 基因(GMFCS I-III;分别为 1、1、5,p<0.04)、COL6 基因(GMFCS I-III;分别为 0、4、7,p<0.003)和 COL9 基因(GMFCS I-III;分别为 1、1、5,p<0.04)在 GMFCS 水平 III 的 CP 患者中的 VUS2 变异明显更多。此外,COL6 基因(GMFCS I-III;分别为 0、5、2,p<0.01)和 COL7 基因(GMFCS I-III;分别为 0、3、0,p<0.04)的 VUS3 变异在 GMFCS 分级 II 级中明显更多。本研究结果强调了 CP 患者中不同胶原类型的多个候选基因变异,这些变异可能具有功能效应。

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