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对摩洛哥散发性肌阵挛-肌张力障碍综合征患者进行SGCE基因突变筛查。

Screening for SGCE mutations in Moroccan sporadic patients with Myoclonus-Dystonia syndrome.

作者信息

Rachad Laila, El Otmani Hicham, Karkar Adnane, El Moutawakil Bouchra, El Kadmiri Nadia, Nadifi Sellama

机构信息

Laboratory of Medical Genetics and Molecular Pathologies, Faculty of Medicine and Pharmacy Hassan II University of Casablanca, Casablanca, Morocco.

Laboratory of Medical Genetics and Molecular Pathologies, Faculty of Medicine and Pharmacy Hassan II University of Casablanca, Casablanca, Morocco; Department of Neurology, IBN ROCHD Universitary Hospital, Casablanca, Morocco.

出版信息

Neurosci Lett. 2019 Jun 11;703:1-4. doi: 10.1016/j.neulet.2019.03.003. Epub 2019 Mar 5.

Abstract

Myoclonus-Dystonia (M-D) is a rare autosomal-dominant movement disorder characterized by myoclonic jerks in combination with dystonia and psychiatric features. Mutations in the Epsilon-sarcoglycan (SGCE, DYT11) gene have been found to cause M-D in 30%-50% of familial M-D. Sporadic cases have also been reported. The aim of study was to investigate whether the M-D phenotype is associated with the existence of SGCE mutations in Moroccan sporadic patients with M-D syndrome. The study included 12 M-D patients. We sequenced the entire coding region of the SGCE gene. We identified two different heterozygous SGCE mutations (c.769A > C ; c.391-3T > C). Our finding confirm that SGCE mutations can occur in sporadic patients when the phenotype is consistent with M-D. Further functional studies are needed to show how changes in SGCE protein function lead to the M-D phenotype.

摘要

肌阵挛性肌张力障碍(M-D)是一种罕见的常染色体显性运动障碍,其特征为肌阵挛性抽搐,并伴有肌张力障碍和精神症状。已发现ε-肌聚糖(SGCE,DYT11)基因突变在30%-50%的家族性M-D中导致发病。也有散发病例的报道。本研究的目的是调查在患有M-D综合征的摩洛哥散发病例中,M-D表型是否与SGCE基因突变的存在有关。该研究纳入了12例M-D患者。我们对SGCE基因的整个编码区进行了测序。我们鉴定出两种不同的杂合SGCE突变(c.769A>C;c.391-3T>C)。我们的发现证实,当表型与M-D一致时,SGCE突变可发生于散发病例中。需要进一步的功能研究来表明SGCE蛋白功能的变化如何导致M-D表型。

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