妊娠肝内胆汁淤积:马耳他队列的单中心全外显子组测序研究。

IF 2.9 3区 医学 Q2 GENETICS & HEREDITY
Dorianne Spiteri, Laura Grech, Charles Savona-Ventura, Nikolai Paul Pace
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引用次数: 0

摘要

妊娠肝内胆汁淤积症(ICP)具有多因素病理生理,涉及遗传、内分泌和环境因素。它与母亲痛苦和不良胎儿结局有关。ICP的单基因病因在马耳他人群中仍未被发现。我们在20个不相关的索引病例中应用全外显子组测序来评估与胆汁酸运输相关的基因变异的分子谱。我们列出了五个独特的杂合变体。检测到三种ABCB4变体,包括一种新的可能致病性停止增益变体。3例家谱不相关的病例携带ABCB4 p.Asn510Ser变异,通过共享单倍型分析提示有一个创始人。这项研究提供了初步的见解,从一个未研究的人群ICP的单基因病因。它扩大了与ICP相关的遗传变异谱,并为马耳他人群中的奠基者效应提供了证据。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Intrahepatic Cholestasis of Pregnancy: A Single-Centre Whole-Exome Sequencing Study in a Maltese Cohort.

Intrahepatic cholestasis of pregnancy (ICP) has a multifactorial pathophysiology involving genetic, endocrine, and environmental factors. It is associated with maternal distress and adverse foetal outcomes. The monogenic aetiology of ICP remains unexplored in the Maltese population. We apply whole exome sequencing in 20 unrelated index cases to assess the molecular spectrum of variants in genes linked to bile acid transport. We shortlisted five unique heterozygous variants. Three ABCB4 variants, including a novel likely pathogenic stop-gain variant were detected. Three genealogically unrelated cases carried an ABCB4 p.Asn510Ser variant, suggestive of a founder through shared haplotype analysis. This study provides preliminary insight into the monogenic aetiology of ICP from an unstudied population. It expands the spectrum of genetic variants associated with ICP and provides evidence for a founder effect in the Maltese population.

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来源期刊
Clinical Genetics
Clinical Genetics 医学-遗传学
CiteScore
6.50
自引率
0.00%
发文量
175
审稿时长
3-8 weeks
期刊介绍: Clinical Genetics links research to the clinic, translating advances in our understanding of the molecular basis of genetic disease for the practising clinical geneticist. The journal publishes high quality research papers, short reports, reviews and mini-reviews that connect medical genetics research with clinical practice. Topics of particular interest are: • Linking genetic variations to disease • Genome rearrangements and disease • Epigenetics and disease • The translation of genotype to phenotype • Genetics of complex disease • Management/intervention of genetic diseases • Novel therapies for genetic diseases • Developmental biology, as it relates to clinical genetics • Social science research on the psychological and behavioural aspects of living with or being at risk of genetic disease
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