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COG6-CDG:扩大表型,重点关注糖基化缺陷在男性性别发育障碍发病机制中的作用。

COG6-CDG: Expanding the phenotype with emphasis on glycosylation defects involved in the causation of male disorders of sex development.

机构信息

Institute of Human Genetics, Galilee Medical Center, Nahariya, Israel.

Azrieli Faculty of Medicine, Bar-Ilan University, Safed, Israel.

出版信息

Clin Genet. 2020 Oct;98(4):402-407. doi: 10.1111/cge.13816. Epub 2020 Aug 4.

Abstract

COG6-congenital disorder of glycosylation (COG6-CDG) is caused by biallelic mutations in COG6. To-date, 12 variants causing COG6-CDG in less than 20 patients have been reported. Using whole exome sequencing we identified two siblings with a novel homozygous deletion of 26 bp in COG6, creating a splicing variant (c.518_540 + 3del) and a shift in the reading frame. The phenotype of COG6-CDG includes growth and developmental retardation, microcephaly, liver and gastrointestinal disease, hypohydrosis and recurrent infections. We report two patients with novel phenotypic features including bowel malrotation and ambiguous genitalia, directing attention to the role of glycoprotein metabolism in the causation of disorders of sex development (DSD). Searching the glycomic literature, we identified 14 CDGs including males with DSD, a feature not previously accentuated. This study broadens the genetic and phenotypic spectrum of COG6-CDG and calls for increasing awareness to the central role of glycosylation processes in development of human sex and genitalia.

摘要

COG6-先天性糖基化障碍(COG6-CDG)是由 COG6 的双等位基因突变引起的。迄今为止,已有 12 种变异导致 COG6-CDG 不到 20 例患者。我们使用全外显子组测序发现了两名同胞存在 COG6 中 26bp 的纯合缺失,导致剪接变异(c.518_540 + 3del)和阅读框移位。COG6-CDG 的表型包括生长和发育迟缓、小头畸形、肝和胃肠道疾病、少汗和反复感染。我们报告了两名具有新表型特征的患者,包括肠旋转不良和生殖器模糊,这提示糖蛋白代谢在性发育障碍(DSD)中的作用。在糖组学文献中搜索,我们发现了 14 种包括男性 DSD 的 CDG,这一特征以前没有被强调。本研究拓宽了 COG6-CDG 的遗传和表型谱,并呼吁提高对糖基化过程在人类性别和生殖器发育中的核心作用的认识。

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