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应用先证者单体外显子组测序对近亲婚配夫妇进行孕前携带者筛查的经验。

Experience using singleton exome sequencing of probands as an approach to preconception carrier screening in consanguineous couples.

机构信息

Department of Clinical and Molecular Genetics, Hospital Universitari Vall d'Hebron, Barcelona, Spain

Medicine Genetics Group Vall d'Hebron Research Institute (VHIR), Barcelona, Spain.

出版信息

J Med Genet. 2023 Jun;60(6):540-546. doi: 10.1136/jmg-2022-108607. Epub 2022 Dec 8.

Abstract

BACKGROUND

Consanguineous couples have an increased risk of severe diseases in offspring due to autosomal recessive disorders. Exome sequencing (ES) offers the possibility of extensive preconception carrier screening (PCS) in consanguineous couples who may be at risk of rare genetic disorders.

METHODS

We retrospectively analysed ES data from 65 probands affected with rare genetic disorders born from consanguineous couples. We explored diagnostic yield and carrier status for recessive disorders.

RESULTS

The overall diagnostic yield in a singleton approach was 53.8%, mostly recessive variants. In a hypothetical exome-based PCS, only 11.7% of these causative rare variants would have been missed in the filtering process. Carrier screening for recessive conditions allowed the identification of at least one additional pathogenic or likely pathogenic variant in 85.7% of the probands, being the majority with a gene carrier frequency <1 in 200. In addition, considering only clinically actionable conditions, we estimated that 12.3% of our close consanguineous couples may be at risk for an additional recessive disease.

CONCLUSIONS

Our results demonstrate that ES outperforms panel-based screening in a PCS context in consanguineous couples and could potentially increase their reproductive autonomy and facilitate informed decision-making.

摘要

背景

由于常染色体隐性疾病,近亲结婚的夫妇生育的后代有患严重疾病的风险。外显子组测序(ES)为可能患有罕见遗传疾病的近亲夫妇提供了广泛的孕前携带者筛查(PCS)的可能性。

方法

我们回顾性分析了 65 名由近亲结婚生育的患有罕见遗传疾病的先证者的 ES 数据。我们探讨了隐性疾病的诊断率和携带者状态。

结果

在单胎方法中,整体诊断率为 53.8%,主要是隐性变异。在假设的基于外显子组的 PCS 中,在过滤过程中仅会错过 11.7% 的这些致病罕见变异。隐性疾病携带者筛查可在 85.7%的先证者中确定至少一个额外的致病性或可能致病性变异,其中大多数基因携带者频率<1/200。此外,仅考虑具有临床可操作性的情况,我们估计 12.3%的近亲夫妇可能面临额外隐性疾病的风险。

结论

我们的结果表明,在外显子组测序在 PCS 背景下优于基于面板的筛查,这可能会增加近亲夫妇的生殖自主权并促进知情决策。

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