Pinar Ozkan Kart, Oguzhan Demir, Ayberk Turkyilmaz, Alper Han Cebi, Ali Cansu
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Three patients from three unrelated families were identified who were comprehensively evaluated with clinical examination, laboratory tests, and imaging studies. The whole exome sequencing (WES) with copy number analysis was performed. The identified variants were confirmed in the proband and parents using Sanger sequencing. Common clinical features of the patients included refractory epileptic seizures, developmental delay, and microcephaly, consistent with the literature. Developmental and Epileptic Encephalopathy starting in the first year of life and burst suppression pattern observed in electroencephalogram are among the important clinical features. WES analysis revealed a homozygous NM_001004127.3: c.953A > C (p. Gln318Pro) missense variant in the ALG11 gene in all three patients. 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引用次数: 0
摘要
先天性糖基化1p型疾病是n -糖基化缺陷之一,天冬酰胺依赖性糖基化11 (ALG11-CDG, #OMIM: 613,666),是一种非常罕见的常染色体隐性糖基化缺陷,可导致多系统受累,经常表现为神经系统症状,如癫痫和神经运动发育迟缓。在这项研究中,我们旨在介绍来自三个不相关家族的三名土耳其患者,他们的临床和基因型发现是ALG11基因复发变异,并将其与文献中描述的其他病例进行比较。来自三个不相关家庭的三名患者被确定,他们通过临床检查、实验室检查和影像学检查进行了全面评估。全外显子组测序(WES)并进行拷贝数分析。使用Sanger测序在先证者和父母中确认鉴定的变异。患者的常见临床特征包括难治性癫痫发作、发育迟缓和小头畸形,与文献一致。发育性和癫痫性脑病开始于生命的第一年,脑电图上观察到的爆发抑制模式是重要的临床特征。WES分析显示,所有3例患者的ALG11基因中均存在纯合子NM_001004127.3: C . 953a > C (p. Gln318Pro)错义变异。本研究介绍了三名土耳其ALG11-CDG亚型患者的临床和遗传特征,该亚型先前已在文献中描述;通过与类似病例的比较,加强了目前对表型多样性的认识。此外,WES在罕见CDG亚型诊断中的作用再次得到证实。
A recurrent c.953A>C (p. Gln318Pro) variant in ALG11 causing congenital disorder of glycosylation in Turkish population.
Congenital disorders of glycosylation type 1p, one of the N-glycosylation defects, Asparagine-dependent glycosylation 11 (ALG11-CDG, #OMIM: 613,666), is a very rare type of autosomal recessive glycosylation defect that causes multisystem involvement and frequently presents with neurological symptoms such as epilepsy and neuromotor developmental delay. In this study, we aimed to present three Turkish patients from three unrelated families with the recurrent variant in the ALG11 gene, along with their clinical and genotypic findings, and to compare them with other cases described in the literature. Three patients from three unrelated families were identified who were comprehensively evaluated with clinical examination, laboratory tests, and imaging studies. The whole exome sequencing (WES) with copy number analysis was performed. The identified variants were confirmed in the proband and parents using Sanger sequencing. Common clinical features of the patients included refractory epileptic seizures, developmental delay, and microcephaly, consistent with the literature. Developmental and Epileptic Encephalopathy starting in the first year of life and burst suppression pattern observed in electroencephalogram are among the important clinical features. WES analysis revealed a homozygous NM_001004127.3: c.953A > C (p. Gln318Pro) missense variant in the ALG11 gene in all three patients. This study presents the clinical and genetic features of three Turkish patients with the ALG11-CDG subtype, which has been previously described in the literature; it reinforces the current knowledge on phenotypic diversity by comparisons with similar cases. In addition, the role of WES in the diagnosis of rare CDG subtypes has been demonstrated once again.
期刊介绍:
Neurogenetics publishes findings that contribute to a better understanding of the genetic basis of normal and abnormal function of the nervous system. Neurogenetic disorders are the main focus of the journal. Neurogenetics therefore includes findings in humans and other organisms that help understand neurological disease mechanisms and publishes papers from many different fields such as biophysics, cell biology, human genetics, neuroanatomy, neurochemistry, neurology, neuropathology, neurosurgery and psychiatry.
All papers submitted to Neurogenetics should be of sufficient immediate importance to justify urgent publication. They should present new scientific results. Data merely confirming previously published findings are not acceptable.