[1]杨莹 陈丽卿 刘艳.儿童COL4A1基因变异相关脑病1例报道并文献复习[J].卒中与神经疾病杂志,2022,29(03):257-261.[doi:10.3969/j.issn.1007-0478.2022.03.013]
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儿童COL4A1基因变异相关脑病1例报道并文献复习()
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《卒中与神经疾病》杂志[ISSN:1007-0478/CN:42-1402/R]

卷:
第29卷
期数:
2022年03期
页码:
257-261
栏目:
论 著
出版日期:
2022-06-25

文章信息/Info

文章编号:
1007-0478(2022)03-0257-05
作者:
杨莹 陈丽卿 刘艳
430030 武汉,华中科技大学同济医学院附属同济医院儿科[杨莹(厦门大学附属妇女儿童医院、厦门市妇幼保健院儿科)陈丽卿 刘艳(通信作者)]
关键词:
COL4A1基因 脑病 小脑蚓部 全外显子测序
分类号:
R742
DOI:
10.3969/j.issn.1007-0478.2022.03.013
文献标志码:
A
摘要:
目的 探讨Ⅳ型胶原蛋白基因α1(α1 type Ⅳ collagen,COL4A1)基因变异相关脑病的临床、影像学表现及基因型特点。方法 收集1例COL4A1基因变异相关脑病患儿的临床资料,并进行全外显子测序,以“COL4A1”、“脑病”为检索词,搜索Pubmed数据库、人类基因组突变数据库(The human gene mutation database,HGMD)检索建库、中国知网及万方医学相关文献,结合既往文献病例总结分析。结果 先证者,女,5岁3月,表现为运动发育迟缓、左侧肢体运动障碍、姿势异常,肌酸肌酶水平增高,头颅磁共振成像(Magnetic resonance imaging,MRI)示双侧小脑形态失常、下蚓部缺如、双侧脑室旁白质病变、基底节软化灶,全外显子测序发现患儿COL4A1基因(NM_001845.5)c.2086G>A(p.Gly696Ser)新生杂合致病性突变。文献检索到87个家庭共152例COL4A1基因变异相关脑病患者,与已报道病例比较,该患儿出现罕见的小脑下蚓部缺如、肢体运动障碍及姿势异常。结论 本例扩大了COL4A1基因变异脑病的临床表现内容,对出现发育迟缓、脑白质病变,尤其是合并眼部、肾脏、肌肉或心脏损害者,应尽早完善COL4A1基因检测。

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更新日期/Last Update: 1900-01-01