[1]史兆博,孙永,耿海威,等.ATP7B基因特殊的复合杂合突变致Wilson病1例报道[J].卒中与神经疾病杂志,2022,29(04):373-376.[doi:10.3969/j.issn.1007-0478.2022.04.014]
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ATP7B基因特殊的复合杂合突变致Wilson病1例报道()
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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04期
页码:
373-376
栏目:
论著
出版日期:
2022-09-10

文章信息/Info

文章编号:
1007-0478(2022)04-0373-04
作者:
史兆博孙永耿海威马丽丽
475000 河南省开封市中心医院[史兆博 孙永 耿海威 马丽丽(通信作者)]
关键词:
ATP7B基因复合杂合突变Wilson病内含子区域启动子区域
分类号:
R742.4
DOI:
10.3969/j.issn.1007-0478.2022.04.014
文献标志码:
A
摘要:
目的 探讨1例ATP7B基因特殊的复合杂合突变导致Wilson病的临床及影像学特征。方法 分析1例疑似Wilson病患者的临床表现及影像学特点,对其进行基因测序。结果 患者14岁开始即出现精神异常,17岁开始出现不自主震颤、写字过小等神经系统症状,血清铜蓝蛋白水平降低,瞳孔笔照射下可见双眼角膜色素环(Kayser-Fleischer ring,K-F环),肝脏彩超提示肝脏弥漫性损害; 具备Wilson病的典型影像学表现,即双侧丘脑、中脑、脑桥对称性长T1长T2信号,轻度脑萎缩改变; 基因检测提示ATP7B基因特殊的复合杂合突变,即c.1470C>A(p.C490*),4号内含子区域剪接突变c.1708-1G>C(splice-3), 启动子区域c.-157-u53A>T, c.1168A>G(p.I390V); 家系验证发现先证者父亲及弟弟存在杂合突变c.1708-1G>C(splice-3),c.-157-u53A>T(promoter), c.1168A>G(p.I390V)。根据美国遗传学与基因组学学会(American college of medical genetics and genomics, ACMG)指南,前3个变异位点均评级为致病性突变,证据分别为PVS1+PM2_支持+PP4; PVS1+PM2_支持+PM3_非常强+PP4; PS3+PM2_支持+PM3_强+PP4。变异位点c.1168A>G评级为临床意义未明(PM2_支持+PP4)。结论 该复合杂合突变患者具备Wilson病典型的临床及影像学特征。

参考文献/References:

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备注/Memo

备注/Memo:
基金项目:开封市科技发展计划项目(1703035)
更新日期/Last Update: 2022-09-10