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中美洲和南美洲家族性帕金森病基因筛查。

Parkinson's Disease Gene Screening in Familial Cases from Central and South America.

机构信息

Veterans Affairs Puget Sound Health Care System, Seattle, Washington, USA.

Department of Neurology, University of Washington School of Medicine, Seattle, Washington, USA.

出版信息

Mov Disord. 2024 Oct;39(10):1843-1855. doi: 10.1002/mds.29931. Epub 2024 Jul 25.

Abstract

BACKGROUND

Parkinson's disease (PD) is the second most common neurodegenerative disease following Alzheimer's disease. Nearly 30 causative genes have been identified for PD and related disorders. However, most of these genes were identified in European-derived families, and little is known about their role in Latin American populations.

OBJECTIVES

Our goal was to assess the spectrum and frequency of pathogenic variants in known PD genes in familial PD patients from Latin America.

METHODS

We selected 335 PD patients with a family history of PD from the Latin American Research Consortium on the Genetics of PD. We capture-sequenced the coding regions of 26 genes related to neurodegenerative parkinsonism. Of the 335 PD patients, 324 had sufficient sequencing coverage to be analyzed.

RESULTS

We identified pathogenic variants in 41 individuals (12.7%) in FBXO7, GCH1, LRRK2, PARK7, PINK1, PLA2G6, PRKN, SNCA, and TARDBP, GBA1 risk variants in 25 individuals (7.7%), and variants of uncertain significance in another 24 individuals (7.4%) in ATP13A2, ATP1A3, DNAJC13, DNAJC6, GBA1, LRKK2, PINK1, VPS13C, and VPS35. Of the 70 unique variants identified, 19 were more frequent in Latin Americans than in any other population.

CONCLUSIONS

This is the first screening of known PD genes in a large cohort of patients with familial PD from Latin America. There were substantial differences in the spectrum of variants observed in comparison to previous findings from PD families of European origin. Our data provide further evidence that differences exist between the genetic architecture of PD in Latinos and European-derived populations. © 2024 The Author(s). Movement Disorders published by Wiley Periodicals LLC on behalf of International Parkinson and Movement Disorder Society.

摘要

背景

帕金森病(PD)是仅次于阿尔茨海默病的第二常见神经退行性疾病。已经确定了近 30 个导致 PD 和相关疾病的致病基因。然而,这些基因大多数是在欧洲血统的家族中发现的,对于它们在拉丁美洲人群中的作用知之甚少。

目的

我们的目标是评估已知 PD 基因在来自拉丁美洲的家族性 PD 患者中的谱和频率。

方法

我们从拉丁美洲帕金森病遗传学研究联盟中选择了 335 名有 PD 家族史的 PD 患者。我们对与神经退行性帕金森病相关的 26 个基因的编码区进行了捕获测序。在 335 名 PD 患者中,有 324 名患者有足够的测序覆盖度可供分析。

结果

我们在 FBXO7、GCH1、LRRK2、PARK7、PINK1、PLA2G6、PRKN、SNCA 和 TARDBP 中发现了 41 名个体(12.7%)的致病性变异,在 GBA1 风险变异中发现了 25 名个体(7.7%),在 ATP13A2、ATP1A3、DNAJC13、DNAJC6、GBA1、LRRK2、PINK1、VPS13C 和 VPS35 中发现了另外 24 名个体(7.4%)的意义不明的变异。在确定的 70 个独特变异中,有 19 个在拉丁美洲比在任何其他人群中更为常见。

结论

这是首次对来自拉丁美洲的大量家族性 PD 患者进行已知 PD 基因的筛查。与之前来自欧洲 PD 家族的发现相比,观察到的变异谱存在很大差异。我们的数据进一步证明了 PD 遗传结构在拉丁美洲人和欧洲裔人群之间存在差异。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/67a0/11490405/ba7a90c230f8/nihms-2007518-f0001.jpg

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