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与原发性小头畸形和胎儿生长受限相关的新型双等位基因 SASS6 变异。

Novel biallelic SASS6 variants associated with primary microcephaly and fetal growth restriction.

机构信息

Department of Medical Genetics and Prenatal Diagnosis, Affiliated Maternity and Child Health Care Hospital of Nantong University, Nantong, China.

Department of Ultrasound, Affiliated Maternity and Child Health Care Hospital of Nantong University, Nantong, China.

出版信息

Am J Med Genet A. 2024 Jul;194(7):e63598. doi: 10.1002/ajmg.a.63598. Epub 2024 Mar 19.

DOI:10.1002/ajmg.a.63598
PMID:38501757
Abstract

Primary microcephaly is characterized by a head circumference prenatally or at birth that falls below three standard deviations from age-, ethnic-, and sex-specific norms. Genetic defects are one of the underlying causes of primary microcephaly. Since 2014, five variants of the SASS6 gene have been identified as the cause of MCPH 14 in three reported families. In this study, we present the genetic findings of members of a nonconsanguineous Chinese couple with a history of microcephaly and fetal growth restriction (FGR) during their first pregnancy. Utilizing trio whole-exome sequencing, we identified compound heterozygous variants involving a frameshift NM_194292.3:c.450_453del p.(Lys150AsnfsTer7) variant and a splice region NM_194292.3:c.1674+3A>G variant within the SASS6 gene in the affected fetus. Moreover, reverse transcriptase-polymerase chain reaction from RNA of the mother's peripheral blood leukocytes revealed that the c.1674+3A>G variant led to the skipping of exon 14 and an inframe deletion. To the best of our knowledge, the association between FGR and SASS6-related microcephaly has not been reported, and our findings confirm the pivotal role of SASS6 in microcephaly pathogenesis and reveal an expanded view of the phenotype and mutation spectrum associated with this gene.

摘要

原发性小头畸形的特征是产前或出生时头围低于年龄、种族和性别特定标准的三个标准差以下。遗传缺陷是原发性小头畸形的一个潜在原因。自 2014 年以来,已经在三个报道的家族中发现了 SASS6 基因的五个变体,这些变体是 MCPH14 的原因。在这项研究中,我们报告了一对非近亲的中国夫妇的遗传发现,他们在第一次怀孕时患有小头畸形和胎儿生长受限(FGR)。利用三体外显子组测序,我们在受影响的胎儿中鉴定出涉及移码 NM_194292.3:c.450_453del p.(Lys150AsnfsTer7)变体和剪接区域 NM_194292.3:c.1674+3A>G 变体的复合杂合变体在 SASS6 基因内。此外,来自母亲外周血白细胞的 RNA 的逆转录-聚合酶链反应显示,c.1674+3A>G 变体导致 14 号外显子跳跃和内含子缺失。据我们所知,FGR 与 SASS6 相关的小头畸形之间的关联尚未报道,我们的发现证实了 SASS6 在小头畸形发病机制中的关键作用,并揭示了与该基因相关的表型和突变谱的扩展观点。

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Am J Med Genet A. 2024 Jul;194(7):e63598. doi: 10.1002/ajmg.a.63598. Epub 2024 Mar 19.
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