18994名种族不同的疑似罕见孟德尔疾病患者的外显子组测序。

IF 4.7 2区 医学 Q1 GENETICS & HEREDITY
Heonjong Han, Go Hun Seo, Seong-In Hyun, Kisang Kwon, Seung Woo Ryu, Rin Khang, Eugene Lee, JiHye Kim, Yongjun Song, Won Chan Jeong, Joohyun Han, Dong-Wook Kim, Soyeon Yang, Sohyun Lee, Sohyun Jang, Jungsul Lee, Hane Lee
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引用次数: 0

摘要

我们研究了外显子组测序(ES)在诊断种族不同的罕见遗传疾病患者中的有效性。对2020 - 2022年间到单一参考实验室就诊的18994例ES患者的诊断率及影响诊断率的因素进行了研究。总诊断率为31.8%。皮肤疾病、骨骼疾病和神经发育疾病的疾病类别、发病年龄早、是否有血缘关系以及是否有亲代测序数据与较高的诊断率相关。我们的数据集中发现了近68K个变体,其频率高于gnomAD 4.0中观察到的频率。其中,507个变异可能被归类为良性,占ClinVar非良性变异的0.04%(507/1,433,904),占我们队列中至少观察到一次的非良性ClinVar变异的0.20%(507/276,777)。总体诊断率与在其他种族背景较少的大型队列研究中观察到的结果相当。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Exome sequencing of 18,994 ethnically diverse patients with suspected rare Mendelian disorders.

We investigated the effectiveness of exome sequencing (ES) in diagnosing ethnically diverse patients with rare genetic disorders. A total of 18,994 patients referred to a single reference laboratory for ES between 2020 and 2022 were studied for the diagnostic rate and factors influencing the diagnostic rate. The overall diagnostic rate was 31.8%. Dermatological disorders, skeletal disorders, and neurodevelopmental disorders disease categories, early age-of-onset, presence of consanguinity, and the presence of parental sequencing data were found to be correlated with a higher diagnostic rate. Nearly 68K variants were identified in our dataset at a higher frequency than that observed in gnomAD 4.0. Of these, 507 variants could be classified as likely benign, representing 0.04% of non-benign variants in ClinVar (507/1,433,904) and 0.20% of the non-benign ClinVar variants observed at least once in our cohort (507/276,777). The overall diagnostic rate is comparable to that observed in other large cohort studies with less diverse ethnic backgrounds.

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来源期刊
NPJ Genomic Medicine
NPJ Genomic Medicine Biochemistry, Genetics and Molecular Biology-Molecular Biology
CiteScore
9.40
自引率
1.90%
发文量
67
审稿时长
17 weeks
期刊介绍: npj Genomic Medicine is an international, peer-reviewed journal dedicated to publishing the most important scientific advances in all aspects of genomics and its application in the practice of medicine. The journal defines genomic medicine as "diagnosis, prognosis, prevention and/or treatment of disease and disorders of the mind and body, using approaches informed or enabled by knowledge of the genome and the molecules it encodes." Relevant and high-impact papers that encompass studies of individuals, families, or populations are considered for publication. An emphasis will include coupling detailed phenotype and genome sequencing information, both enabled by new technologies and informatics, to delineate the underlying aetiology of disease. Clinical recommendations and/or guidelines of how that data should be used in the clinical management of those patients in the study, and others, are also encouraged.
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