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以小脑共济失调、神经病变和前庭反射消失为表现的新型DNMT1突变

Cerebellar Ataxia, Neuropathy, and Vestibular Areflexia as a Presentation of a Novel DNMT1 Mutation.

作者信息

Sossamon Jake, O'Connell Patrick, Rastall David P W

机构信息

Medical University of South Carolina College of Medicine, 171 Ashley Ave, Charleston, SC, USA.

The Icahn School of Medicine at Mount Sinai, Mount Sinai Kravis Children's Hospital, 1184 5th Ave, New York, NY, USA.

出版信息

Cerebellum. 2025 Jan 4;24(1):25. doi: 10.1007/s12311-024-01780-0.

Abstract

A 50-year-old woman with a 20-year history of gait instability presented with new-onset vertigo and oscillopsia. Examination revealed bilateral vestibular loss, cerebellar ataxia, sensory neuropathy, a "yes-yes" head tremor, nystagmus and a family history of a similar syndrome. Genetic testing for cerebellar ataxia with neuropathy and bilateral vestibular areflexia syndrome (RFC1) was negative, but whole exome sequencing identified a novel mutation in the DNA methyltransferase 1 (DNMT1) gene, broadening the differential diagnosis for this phenotype. Management was focused on symptomatic treatment and genetic counseling. This case highlights the importance of considering DNMT1 mutations in patients with a similar clinical presentation.

摘要

一名有20年步态不稳病史的50岁女性出现新发眩晕和视振荡。检查发现双侧前庭功能丧失、小脑共济失调、感觉神经病变、“yes-yes”型头部震颤、眼球震颤以及类似综合征的家族史。针对伴有神经病变和双侧前庭无反射综合征(RFC1)的小脑共济失调进行的基因检测结果为阴性,但全外显子组测序在DNA甲基转移酶1(DNMT1)基因中发现了一个新突变,拓宽了对该表型的鉴别诊断范围。治疗重点是对症治疗和遗传咨询。该病例突出了对于具有相似临床表现的患者考虑DNMT1突变的重要性。

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