基因组测序在一个队列32胎儿遗传骨骼疾病。

IF 4.6 2区 生物学 Q2 BIOCHEMISTRY & MOLECULAR BIOLOGY
Hillevi Lindelöf, Anna Hammarsjö, Ulrika Voss, Serena Gaetana Piticchio, Peter Conner, Nikos Papadogiannakis, Dominyka Batkovskyte, Laura Orellana, Malin Kvarnung, Helena Malmgren, Kristina Lagerstedt Robinson, Ann Nordgren, Anna Lindstrand, Gen Nishimura, Giedre Grigelioniene
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引用次数: 0

摘要

大约200种遗传性骨骼疾病可以在产前出现,通过怀孕期间的超声异常检测到。严重的形式通常在妊娠早期或中期被发现,而较轻的表型在妊娠晚期被发现。由于大量的疾病和重叠的临床表现可以通过超声检测,产前诊断骨骼发育不良是具有挑战性的。该研究于2015年至2022年在卡罗林斯卡大学医院进行,检查了32名胎儿(14名女性和18名男性,来自无血缘关系的家庭)的遗传和x线检查结果,这些胎儿在产前超声检查中发现骨骼异常,并在出生时或终止妊娠后通过x线检查证实。所有32个胎儿的胎儿DNA样本使用硅骨发育不良基因面板进行单胎基因组测序。作为第二步,对6个分子诊断未解的胎儿,进行了涉及胎儿和父母双方的三人基因组测序分析。基因组测序的诊断率为72%,在32个胎儿中发现了23个致病或可能致病的变异。此外,在四个常染色体隐性遗传病的胎儿中发现了四个不确定意义的变异,根据临床和放射学特征以及结构蛋白分析,强烈怀疑是致病的。五种胎儿的分子诊断仍未得到解决。总之,通过将详细的表型数据与单例基因组测序相结合,我们能够对卡罗林斯卡大学医院调查的32例胎儿遗传性骨骼疾病病例中的72%进行遗传诊断。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Genome sequencing in a cohort of 32 fetuses with genetic skeletal disorders.

Approximately 200 genetic skeletal disorders can present prenatally, detectable through ultrasound abnormalities during pregnancy. Severe forms are typically identified during the first or second trimester, whereas milder phenotypes are recognized later, in the third trimester. Diagnosing skeletal dysplasia prenatally is challenging due to the large number of disorders and the overlapping clinical findings that can be detected by ultrasound. This study, conducted at Karolinska University Hospital between 2015 and 2022, examines the genetic and radiographic findings in 32 fetuses (14 female and 18 male, from unrelated families) with skeletal abnormalities detected on prenatal ultrasound and confirmed by radiographs at birth or after pregnancy termination. Fetal DNA samples from all 32 fetuses underwent singleton genome sequencing using an in silico skeletal dysplasia gene panel. As a second step, for six fetuses with molecularly unsolved diagnoses, trio genome sequencing analysis involving the fetus and both parents was performed. The diagnostic yield of genome sequencing was 72%, with pathogenic or likely pathogenic variants identified in 23 of the 32 fetuses. Additionally, four variants of uncertain significance, strongly suspected to be causative based on clinical and radiographic features, as well as structural protein analyses, were identified in four fetuses with autosomal recessive conditions. The diagnoses of five fetuses remain molecularly unsolved. In conclusion, by combining detailed phenotypic data with singleton genome sequencing we were able to reach a genetic diagnosis in 72% of 32 fetal genetic skeletal disorder cases investigated at the Karolinska University Hospital.

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来源期刊
European Journal of Human Genetics
European Journal of Human Genetics 生物-生化与分子生物学
CiteScore
9.90
自引率
5.80%
发文量
216
审稿时长
2 months
期刊介绍: The European Journal of Human Genetics is the official journal of the European Society of Human Genetics, publishing high-quality, original research papers, short reports and reviews in the rapidly expanding field of human genetics and genomics. It covers molecular, clinical and cytogenetics, interfacing between advanced biomedical research and the clinician, and bridging the great diversity of facilities, resources and viewpoints in the genetics community. Key areas include: -Monogenic and multifactorial disorders -Development and malformation -Hereditary cancer -Medical Genomics -Gene mapping and functional studies -Genotype-phenotype correlations -Genetic variation and genome diversity -Statistical and computational genetics -Bioinformatics -Advances in diagnostics -Therapy and prevention -Animal models -Genetic services -Community genetics
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