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在高度近亲结婚的人群中,胎儿结构畸形的产前外显子组测序和染色体微阵列分析揭示了纤毛病基因导致多系统表型的倾向。

Prenatal exome sequencing and chromosomal microarray analysis in fetal structural anomalies in a highly consanguineous population reveals a propensity of ciliopathy genes causing multisystem phenotypes.

机构信息

Centre for Genomic Medicine, King Faisal Specialist Hospital and Research Centre, MBC# 26, P. O. Box 3354, Riyadh, Saudi Arabia.

Saudi Diagnostics Laboratory, KFSHI, P.O.BOX 6802, Riyadh, 12311, Saudi Arabia.

出版信息

Hum Genet. 2022 Jan;141(1):101-126. doi: 10.1007/s00439-021-02406-9. Epub 2021 Dec 1.

Abstract

Fetal abnormalities are detected in 3% of all pregnancies and are responsible for approximately 20% of all perinatal deaths. Chromosomal microarray analysis (CMA) and exome sequencing (ES) are widely used in prenatal settings for molecular genetic diagnostics with variable diagnostic yields. In this study, we aimed to determine the diagnostic yield of trio-ES in detecting the cause of fetal abnormalities within a highly consanguineous population. In families with a history of congenital anomalies, a total of 119 fetuses with structural anomalies were recruited and DNA from invasive samples were used together with parental DNA samples for trio-ES and CMA. Data were analysed to determine possible underlying genetic disorders associated with observed fetal phenotypes. The cohort had a known consanguinity of 81%. Trio-ES led to diagnostic molecular genetic findings in 59 fetuses (with pathogenic/likely pathogenic variants) most with multisystem or renal abnormalities. CMA detected chromosomal abnormalities compatible with the fetal phenotype in another 7 cases. Monogenic ciliopathy disorders with an autosomal recessive inheritance were the predominant cause of multisystem fetal anomalies (24/59 cases, 40.7%) with loss of function variants representing the vast majority of molecular genetic abnormalities. Heterozygous de novo pathogenic variants were found in four fetuses. A total of 23 novel variants predicted to be associated with the phenotype were detected. Prenatal trio-ES and CMA detected likely causative molecular genetic defects in a total of 55% of families with fetal anomalies confirming the diagnostic utility of trio-ES and CMA as first-line genetic test in the prenatal diagnosis of multisystem fetal anomalies including ciliopathy syndromes.

摘要

胎儿异常在所有妊娠中的检出率为 3%,约占所有围产儿死亡的 20%。染色体微阵列分析(CMA)和外显子组测序(ES)广泛应用于产前分子遗传学诊断,具有不同的诊断率。本研究旨在确定三核苷酸 ES 在高度近亲繁殖人群中检测胎儿异常病因的诊断率。在有先天性畸形病史的家庭中,共招募了 119 例有结构异常的胎儿,并对侵入性样本的 DNA 以及父母的 DNA 样本进行三核苷酸 ES 和 CMA。对数据进行分析,以确定与观察到的胎儿表型相关的潜在遗传疾病。该队列的已知近亲率为 81%。三核苷酸 ES 导致 59 例胎儿(有致病性/可能致病性变异)出现诊断性分子遗传学发现,大多数伴有多系统或肾脏异常。CMA 还在另外 7 例病例中检测到与胎儿表型相符的染色体异常。常染色体隐性遗传的单基因纤毛病是多系统胎儿畸形的主要原因(24/59 例,40.7%),绝大多数分子遗传异常是功能丧失变异。在 4 例胎儿中发现杂合性新生致病性变异。共检测到 23 个预测与表型相关的新变异。三核苷酸 ES 和 CMA 总共在 55%的胎儿畸形家庭中检测到可能的致病分子遗传缺陷,证实了三核苷酸 ES 和 CMA 作为产前诊断多系统胎儿畸形(包括纤毛病综合征)的一线基因检测的诊断效用。

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