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新型突变 p.Asp251Asn 位于琥珀酰辅酶 A 连接酶的β亚基,可导致脑肌病和琥珀酰肉碱水平升高。

The novel mutation p.Asp251Asn in the β-subunit of succinate-CoA ligase causes encephalomyopathy and elevated succinylcarnitine.

机构信息

School of Biology, College of Science, University of Tehran, Tehran, Iran.

出版信息

J Hum Genet. 2013 Aug;58(8):526-30. doi: 10.1038/jhg.2013.45. Epub 2013 Jun 13.

Abstract

SUCLA2 is one of several nuclear-encoded genes that can cause encephalomyopathy accompanied by mitochondrial DNA depletion. The disorder usually manifests in early childhood and leads to early death. The gene encodes one of the subunits of succinyl-CoA synthase, the enzyme that catalyzes the reversible conversion of substrates succinyl-CoA and ADP to products succinate and ATP in the tricarboxylic acid pathway. Thirty-two individuals harboring mutations in SUCLA2 have so far been reported, and five different mutations were observed among these individuals. Here we report identification of a novel mutation in SUCLA2 in two cousins affected with encephalomyopathy. The novel mutation causes p.Asp251Asn; the affected amino acid is likely positioned within the ATP-grasp domain of the encoded protein. As previously reported in other patients, we did not observe elevation of methylmalonic acid, the biochemical hallmark of patients with mutations in SUCLA2. We instead found elevated levels of succinylcarnitine.

摘要

SUCLA2 是几个核编码基因之一,这些基因可导致伴线粒体 DNA 耗竭的脑肌病。该疾病通常在儿童早期发病,导致早亡。该基因编码琥珀酰辅酶 A 合酶的一个亚基,该酶在三羧酸途径中催化琥珀酰辅酶 A 和 ADP 可逆转化为琥珀酸和 ATP。迄今为止,已有 32 名个体携带有 SUCLA2 突变,这些个体中观察到了 5 种不同的突变。在此,我们报道了两位表兄弟患有脑肌病,他们的 SUCLA2 中存在新的突变。该新突变导致 p.Asp251Asn;受影响的氨基酸可能位于编码蛋白的 ATP 捕捉结构域内。与其他患者的先前报道一样,我们未观察到甲基丙二酸水平升高,而甲基丙二酸是 SUCLA2 基因突变患者的生化特征。相反,我们发现琥珀酰肉碱水平升高。

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