Suppr超能文献

由 SATB2 下游染色体断裂区引起的玻璃综合征。

Glass syndrome derived from chromosomal breakage downstream region of SATB2.

机构信息

Department of Transfusion Medicine and Cell Processing, Tokyo Women's Medical University, Tokyo, Japan; Institute of Medical Genetics, Tokyo Women's Medical University, Tokyo, Japan; Division of Gene Medicine, Graduate School of Medicine, Tokyo Women's Medical University, Tokyo, Japan.

Division of Gene Medicine, Graduate School of Medicine, Tokyo Women's Medical University, Tokyo, Japan; Department of Pediatrics, Tokyo Women's Medical University, Tokyo, Japan.

出版信息

Brain Dev. 2024 Oct;46(9):281-285. doi: 10.1016/j.braindev.2024.06.003. Epub 2024 Jul 6.

Abstract

BACKGROUND

Glass syndrome, derived from chromosomal 2q33.1 microdeletions, manifests with intellectual disability, microcephaly, epilepsy, and distinctive features, including micrognathia, down-slanting palpebral fissures, cleft palate, and crowded teeth. Recently, SATB2 located within the deletion region, was identified as the causative gene responsible for Glass syndrome. Numerous disease-causing variants within the SATB2 coding region have been reported.

OBJECTIVE

Given the presentation of intellectual disability and multiple congenital anomalies in a patient with a de novo reciprocal translocation between chromosomes 1 and 2, disruption of the causative gene(s) was suspected. This study sought to identify the causative gene in the patient.

METHODS

Long-read whole-genome sequencing was performed, and the expression level of the candidate gene was analyzed.

RESULTS

The detection of breakpoints was successful. While the breakpoint on chromosome 1 disrupted RNF220, it was not deemed to be a genetic cause. Conversely, SATB2 is located in the approximately 100-kb telomeric region of the breakpoint on chromosome 2. The patient's clinical features resembled those of previously reported cases of Glass syndrome, despite the lack of confirmed reduced SATB2 expression.

CONCLUSION

The patient was diagnosed with Glass syndrome due to the similarity in clinical features. This led us to hypothesize that disruption in the downstream region of SATB2 could result in Glass syndrome. The microhomologies identified in the breakpoint junctions indicate a potential molecular mechanism involving microhomology-mediated break-induced repair mechanism or template switching.

摘要

背景

玻璃综合征源自于染色体 2q33.1 微缺失,表现为智力障碍、小头畸形、癫痫和独特的特征,包括小下颌、下斜的睑裂、腭裂和牙齿拥挤。最近,位于缺失区域内的 SATB2 被鉴定为导致玻璃综合征的致病基因。在 SATB2 编码区域内已经报道了许多致病变异。

目的

鉴于患者存在智力障碍和多种先天性异常,且染色体 1 和 2 之间存在新发相互易位,怀疑致病基因受到了干扰。本研究旨在鉴定该患者的致病基因。

方法

进行长读长全基因组测序,并分析候选基因的表达水平。

结果

成功检测到断点。虽然染色体 1 上的断点破坏了 RNF220,但它不被认为是遗传原因。相反,SATB2 位于染色体 2 上的大约 100kb 端粒区域的断点内。尽管未确认 SATB2 表达减少,但患者的临床特征与先前报道的玻璃综合征病例相似。

结论

由于临床特征相似,该患者被诊断为玻璃综合征。这使我们假设 SATB2 下游区域的破坏可能导致玻璃综合征。在断点连接处鉴定出的微同源序列表明存在一种潜在的分子机制,涉及微同源介导的断裂诱导修复机制或模板转换。

文献AI研究员

20分钟写一篇综述,助力文献阅读效率提升50倍。

立即体验

用中文搜PubMed

大模型驱动的PubMed中文搜索引擎

马上搜索

文档翻译

学术文献翻译模型,支持多种主流文档格式。

立即体验