Suppr超能文献

一个患有帕金森病、特发性震颤、贝尔麻痹且存在帕金基因突变的家族。

A family with Parkinson disease, essential tremor, bell palsy, and parkin mutations.

作者信息

Deng Hao, Le Wei-Dong, Hunter Christine B, Mejia Nicte, Xie Wen-Jie, Jankovic Joseph

机构信息

Department of Neurology, Baylor College of Medicine, Houston, TX 77030, USA.

出版信息

Arch Neurol. 2007 Mar;64(3):421-4. doi: 10.1001/archneur.64.3.421.

Abstract

BACKGROUND

Mutations in the parkin gene cause autosomal recessive early-onset Parkinson disease (EOPD). The A265G variant in the HS1 binding protein 3 gene (HS1BP3) is common in essential tremor (ET).

OBJECTIVE

To investigate the presence of mutations in the parkin gene and the A265G variant in the HS1BP3 gene in a Mexican family with EOPD, ET, and Bell palsy.

DESIGN

Direct sequencing, semiquantitative polymerase chain reaction, and reverse transcription-polymerase chain reaction were performed in the 14 members of this family.

SETTING

Mexican family. Patients Two patients with EOPD were analyzed.

RESULTS

Compound heterozygous mutations (EX 3_6 del and EX 5 del) in the parkin gene were identified in 2 patients with EOPD, characterized by beneficial response to levodopa, relatively slow progression, and motor complications. Although heterozygous EX 3_6 del and homozygous EX 5 del mutations in the parkin gene have been previously described, to our knowledge, this is the first report of these mutations in compound heterozygotes. Seven heterozygous A265G variants in the HS1BP3 gene were found in this pedigree, but they did not cosegregate with ET, Parkinson disease, or Bell palsy, supporting the conclusion that this variant is not associated with ET.

CONCLUSIONS

Compound heterozygous parkin mutations (EX 3_6 del and EX 5 del) caused EOPD in this family, but the A265G variant in the HS1BP3 gene, previously considered to be responsible for ET, was probably not pathogenically related to the ET in this family.

摘要

背景

帕金森病基因(parkin)突变会导致常染色体隐性早发性帕金森病(EOPD)。HS1结合蛋白3基因(HS1BP3)中的A265G变异在特发性震颤(ET)中很常见。

目的

在一个患有EOPD、ET和贝尔麻痹的墨西哥家族中,研究帕金森病基因的突变情况以及HS1BP3基因中的A265G变异。

设计

对该家族的14名成员进行直接测序、半定量聚合酶链反应和逆转录-聚合酶链反应。

地点

墨西哥家族。患者 分析了两名EOPD患者。

结果

在两名EOPD患者中鉴定出帕金森病基因的复合杂合突变(EX 3_6缺失和EX 5缺失),其特征为对左旋多巴反应良好、进展相对缓慢且有运动并发症。尽管此前已描述过帕金森病基因中的杂合EX 3_6缺失和纯合EX 5缺失突变,但据我们所知,这是这些突变在复合杂合子中的首次报道。在这个家系中发现了7个HS1BP3基因的杂合A265G变异,但它们与ET、帕金森病或贝尔麻痹没有共分离,支持该变异与ET无关的结论。

结论

复合杂合帕金森病基因突变(EX 3_6缺失和EX 5缺失)在这个家族中导致了EOPD,但HS1BP3基因中的A265G变异(此前被认为与ET有关)可能与该家族中的ET没有致病相关性。

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