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一名患有癫痫、发育迟缓及明显畸形特征的女性,其MBD5基因存在2q23.1微缺失。

2q23.1 microdeletion of the MBD5 gene in a female with seizures, developmental delay and distinct dysmorphic features.

作者信息

Noh Grace J, Graham John M

机构信息

Medical Genetics Institute, Cedars-Sinai Medical Center, 8700 Beverly Blvd, PACT, Suite 400, Los Angeles, CA 90048, USA.

出版信息

Eur J Med Genet. 2012 Jan;55(1):59-62. doi: 10.1016/j.ejmg.2011.10.001. Epub 2011 Oct 24.

Abstract

We report a 2-year-old female who initially presented with seizures, developmental delay and dysmorphic features and was found to have a 0.3 Mb deletion at chromosome 2q23.1 encompassing the critical seizure gene, MBD5. Her distinct physical features include bifrontal narrowing with brachycephaly, low anterior hairline, hypotonic facial features with short upturned nose, flat nasal bridge, hypertelorism, tented upper lip with everted lower lip, downturned corners of her mouth, and relatively coarse facial features including thickened tongue. She also had a short neck, brachytelephalangy, clinodactyly, and hypertrichosis. At 3½ years she developed progressive ataxia and lost vocabulary at the age of 4. Regression has been reported in one other case of MBD5 deletion. MBD5 is a member of the methyl binding gene family and appears to be responsible for regulating DNA methylation in the central nervous system. Our patient was entirely deleted for the MBD5 gene with partial loss of the EPC2 gene, which suggests that haploinsufficiency of MBD5 is responsible for the distinct phenotype observed. This supports the hypothesis that MBD5 is indeed the critical gene implicated for the findings seen in patients with deletions of chromosome 2q23.1. Further studies are necessary to delineate the role that the MBD5 gene plays in the development of the brain and these specific physical characteristics.

摘要

我们报告了一名2岁女性,她最初表现为癫痫发作、发育迟缓及畸形特征,被发现2号染色体q23.1处有一个0.3 Mb的缺失,该区域包含关键癫痫基因MBD5。她独特的身体特征包括双额变窄伴短头畸形、前发际线低、面部肌张力低下,短而上翘的鼻子、鼻梁扁平、眼距过宽、上唇呈帐篷状伴下唇外翻、嘴角下垂,以及包括舌头增厚在内的相对粗糙的面部特征。她还患有短颈、短指畸形、小指内弯和多毛症。3岁半时她出现进行性共济失调,4岁时词汇量减少。另一例MBD5缺失病例也有发育倒退的报道。MBD5是甲基结合基因家族的成员之一,似乎负责调节中枢神经系统中的DNA甲基化。我们的患者MBD5基因完全缺失,EPC2基因部分缺失,这表明MBD5单倍体不足是所观察到的独特表型的原因。这支持了MBD5确实是与2号染色体q23.1缺失患者所见发现相关的关键基因这一假说。需要进一步研究来阐明MBD5基因在大脑发育和这些特定身体特征中所起的作用。

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