Suppr超能文献

在三个葡萄糖-6-磷酸脱氢酶严重缺乏症的家族中发现了一种新的 G6PD 有害变异体。

A novel G6PD deleterious variant identified in three families with severe glucose-6-phosphate dehydrogenase deficiency.

机构信息

Department of Clinical Laboratory, Renmin Hospital of Wuhan University, 99 Ziyang Road of Wuchang District, Wuhan, 430060, People's Republic of China.

Department of Pathology, Affiliated Tianyou Hospital of Wuhan University of Science and Technology, Wuhan, 430064, People's Republic of China.

出版信息

BMC Med Genet. 2020 Jul 17;21(1):150. doi: 10.1186/s12881-020-01090-2.

Abstract

BACKGROUND

Glucose-6-phosphate dehydrogenase deficiency (D-G6PD) is an X-linked recessive disorder resulted from deleterious variants in the housekeeping gene Glucose-6-phosphate 1-dehydrogenase (G6PD), causing impaired response to oxidizing agents. Screening for new variations of the gene helps with early diagnosis of D-G6PD resulting in a reduction of disease related complications and ultimately increased life expectancy of the patients.

METHODS

One thousand five hundred sixty-five infants with pathological jaundice were screened for G6PD variants by Sanger sequencing all of the 13 exons, and the junctions of exons and introns of the G6PD gene.

RESULTS

We detected G6PD variants in 439 (28.1%) of the 1565 infants with pathological jaundice. In total, 9 types of G6PD variants were identified in our cohort; and a novel G6PD missense variant c.1118 T > C, p.Phe373Ser in exon 9 of the G6PD gene was detected in three families. Infants with this novel variant showed decreased activity of G6PD, severe anemia, and pathological jaundice, consistent with Class I G6PD deleterious variants. Analysis of the resulting protein's structure revealed this novel variant affects G6PD protein stability, which could be responsible for the pathogenesis of D-G6PD in these patients.

CONCLUSIONS

High rates of G6PD variants were detected in infants with pathological jaundice, and a novel Class I G6PD deleterious variants was identified in our cohort. Our data reveal that variant analysis is helpful for the diagnosis of D-G6PD in patients, and also for the expansion of the spectrum of known G6PD variants used for carrier detection and prenatal diagnosis.

摘要

背景

葡萄糖-6-磷酸脱氢酶缺乏症(D-G6PD)是一种 X 连锁隐性遗传病,由管家基因葡萄糖-6-磷酸 1-脱氢酶(G6PD)中的有害变异引起,导致对氧化剂的反应受损。对该基因的新变异进行筛查有助于早期诊断 D-G6PD,从而减少与疾病相关的并发症,并最终提高患者的预期寿命。

方法

通过对 13 个外显子和外显子与内含子的连接处进行 Sanger 测序,对 1565 例病理性黄疸婴儿进行 G6PD 变异筛查。

结果

我们在 1565 例病理性黄疸婴儿中检测到 439 例(28.1%)G6PD 变异。在我们的队列中总共发现了 9 种 G6PD 变异;并在三个家庭中检测到 G6PD 基因外显子 9 中 c.1118 T > C,p.Phe373Ser 的新型 G6PD 错义变异。具有这种新型变异的婴儿表现出 G6PD 活性降低、严重贫血和病理性黄疸,与 I 类 G6PD 有害变异一致。对产生的蛋白质结构的分析表明,这种新型变异影响 G6PD 蛋白质的稳定性,这可能是导致这些患者发生 D-G6PD 的发病机制。

结论

在病理性黄疸婴儿中检测到高比例的 G6PD 变异,并且在我们的队列中发现了一种新型的 I 类 G6PD 有害变异。我们的数据表明,变异分析有助于对患者的 D-G6PD 进行诊断,也有助于扩展已知 G6PD 变异的谱,用于携带者检测和产前诊断。

文献检索

告别复杂PubMed语法,用中文像聊天一样搜索,搜遍4000万医学文献。AI智能推荐,让科研检索更轻松。

立即免费搜索

文件翻译

保留排版,准确专业,支持PDF/Word/PPT等文件格式,支持 12+语言互译。

免费翻译文档

深度研究

AI帮你快速写综述,25分钟生成高质量综述,智能提取关键信息,辅助科研写作。

立即免费体验