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土耳其婴儿严重肌阵挛癫痫(SMEI)患者中的四种新型SCN1A突变。

Four novel SCN1A mutations in Turkish patients with severe myoclonic epilepsy of infancy (SMEI).

作者信息

Arlier Zulfikar, Bayri Yasar, Kolb Luis E, Erturk Ozdem, Ozturk Ali K, Bayrakli Fatih, Bilguvar Kaya, Moliterno Jennifer A, Dervent Aysin, Demirbilek Veysi, Yalcinkaya Cengiz, Korkmaz Baris, Tuysuz Beyhan, Gunel Murat

机构信息

Department of Neurosurgery, Yale University School of Medicine, New Haven, CT 06510, USA.

出版信息

J Child Neurol. 2010 Oct;25(10):1265-8. doi: 10.1177/0883073809357241. Epub 2010 Jan 28.

Abstract

Severe myoclonic epilepsy of infancy (SMEI) (OMIM #607208), also known as Dravet syndrome, is a rare genetic disorder characterized by frequent generalized, unilateral clonic or tonic-clonic seizures that begin during the first year of life. Heterozygous de novo mutations in the SCN1A gene, which encodes the neuronal voltage-gated sodium channel α subunit type 1 (Nav1.1), are responsible for Dravet syndrome, with a broad spectrum of mutations and rearrangements having been reported. In this study, the authors present 4 novel mutations and confirm 2 previously identified mutations in the SCN1A gene found in a cohort of Turkish patients with Dravet syndrome. Mutational analysis of other responsible genes, GABRG2 and PCDH19, were unrevealing. The authors' findings add to the known spectrum of mutations responsible for this disease phenotype and once again reinforce our understanding of the allelic heterogeneity of this disease.

摘要

婴儿严重肌阵挛癫痫(SMEI)(OMIM #607208),也称为德拉韦特综合征,是一种罕见的遗传性疾病,其特征为在生命的第一年开始出现频繁的全身性、单侧阵挛或强直阵挛发作。编码神经元电压门控钠通道α1亚基(Nav1.1)的SCN1A基因中的杂合新生突变是德拉韦特综合征的病因,已有广泛的突变和重排报道。在本研究中,作者报告了4个新突变,并在一组土耳其德拉韦特综合征患者中证实了先前在SCN1A基因中鉴定出的2个突变。对其他致病基因GABRG2和PCDH19的突变分析未发现异常。作者的发现增加了已知的导致该疾病表型的突变谱,并再次强化了我们对该疾病等位基因异质性的理解。

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