不同人群中重复扩展突变的频率增加

IF 58.7 1区 医学 Q1 BIOCHEMISTRY & MOLECULAR BIOLOGY
Kristina Ibañez, Bharati Jadhav, Matteo Zanovello, Delia Gagliardi, Christopher Clarkson, Stefano Facchini, Paras Garg, Alejandro Martin-Trujillo, Scott J. Gies, Valentina Galassi Deforie, Anupriya Dalmia, Davina J. Hensman Moss, Jana Vandrovcova, Clarissa Rocca, Loukas Moutsianas, Chiara Marini-Bettolo, Helen Walker, Chris Turner, Maryam Shoai, Jeffrey D. Long, Pietro Fratta, Douglas R. Langbehn, Sarah J. Tabrizi, Mark J. Caulfield, Andrea Cortese, Valentina Escott-Price, John Hardy, Henry Houlden, Andrew J. Sharp, Arianna Tucci
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引用次数: 0

摘要

重复扩展障碍(REDs)是一组以神经系统疾病为主的破坏性疾病。这些疾病很常见,全球每 3,000 人中就有 1 人患病,但因人群而异。然而,由于临床表现不尽相同、地理分布各异以及技术限制,REDs 的患病率估算受到了阻碍,导致估算不足。在此,我们利用来自不同人群的 82176 个个体的全基因组测序数据,发现 REDs 的总体疾病等位基因频率为每 283 个个体中有 1 个。我们利用基因数据、发病年龄和存活率对疾病流行率进行建模,结果表明,REDs 患者的预期人数将比目前报告的数字高出两到三倍,这表明诊断不足和/或渗透不完全。虽然有些 REDs 具有人群特异性,例如非洲人中的亨廷顿病 2 型,但大多数 REDs 在所有广泛的遗传血统(即欧洲人、非洲人、美国人、东亚人和南亚人)中都有体现,这对某些 REDs 只出现在特定人群中的观点提出了质疑。这些结果对当地和全球卫生界诊断和咨询 REDs 具有世界性的影响。
本文章由计算机程序翻译,如有差异,请以英文原文为准。

Increased frequency of repeat expansion mutations across different populations

Increased frequency of repeat expansion mutations across different populations

Repeat expansion disorders (REDs) are a devastating group of predominantly neurological diseases. Together they are common, affecting 1 in 3,000 people worldwide with population-specific differences. However, prevalence estimates of REDs are hampered by heterogeneous clinical presentation, variable geographic distributions and technological limitations leading to underascertainment. Here, leveraging whole-genome sequencing data from 82,176 individuals from different populations, we found an overall disease allele frequency of REDs of 1 in 283 individuals. Modeling disease prevalence using genetic data, age at onset and survival, we show that the expected number of people with REDs would be two to three times higher than currently reported figures, indicating underdiagnosis and/or incomplete penetrance. While some REDs are population specific, for example, Huntington disease-like 2 in Africans, most REDs are represented in all broad genetic ancestries (that is, Europeans, Africans, Americans, East Asians and South Asians), challenging the notion that some REDs are found only in specific populations. These results have worldwide implications for local and global health communities in the diagnosis and counseling of REDs.

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来源期刊
Nature Medicine
Nature Medicine 医学-生化与分子生物学
CiteScore
100.90
自引率
0.70%
发文量
525
审稿时长
1 months
期刊介绍: Nature Medicine is a monthly journal publishing original peer-reviewed research in all areas of medicine. The publication focuses on originality, timeliness, interdisciplinary interest, and the impact on improving human health. In addition to research articles, Nature Medicine also publishes commissioned content such as News, Reviews, and Perspectives. This content aims to provide context for the latest advances in translational and clinical research, reaching a wide audience of M.D. and Ph.D. readers. All editorial decisions for the journal are made by a team of full-time professional editors. Nature Medicine consider all types of clinical research, including: -Case-reports and small case series -Clinical trials, whether phase 1, 2, 3 or 4 -Observational studies -Meta-analyses -Biomarker studies -Public and global health studies Nature Medicine is also committed to facilitating communication between translational and clinical researchers. As such, we consider “hybrid” studies with preclinical and translational findings reported alongside data from clinical studies.
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