Qu Jiangbo, Zhu Wenjuan, Wang Ju, Gao Lu, Yu Dongyi
Center for Medical Genetics and Prenatal Diagnosis of Shandong Maternal and Child Health Care Hospital (Key Laboratory of Birth Defect Prevention and Genetic Medicine of Shandong Provincial Health Commission, Key Laboratory of Maternal and Fetal Medicine of National Health Commission of China), Jinan, Shandong 250014, China.
Zhonghua Yi Xue Yi Chuan Xue Za Zhi. 2025 Apr 10;42(4):446-453. doi: 10.3760/cma.j.cn511374-20240716-00393.
To explore the genetic etiology of two fetuses with Mosaic variegated aneuploidy syndrome (MVA) in a pedigree.
A 30-year-old pregnant woman, who presented at the Center for Medical Genetics and Prenatal Diagnosis of Shandong Maternal and Child Health Care Hospital on November 16, 2023, was enrolled. Clinical data of the pedigree were collected, and peripheral blood samples from the parents and amniotic fluid samples from the two fetuses were obtained for genomic DNA extraction. Whole exome sequencing (WES) was performed on both fetuses, followed by Sanger sequencing for familial validation and pathogenicity analysis of candidate variants. Chromosomal karyotyping of the parents was conducted to quantify the proportion of premature chromatid separation (PCS). This study was approved by the Medical Ethics Committee of Shandong Maternal and Child Health Care Hospital (Ethics No. 2024-034).
Both fetuses exhibited structural brain anomalies and developmental delays during the second trimester. Amniocyte karyotyping revealed low-level mosaic aneuploidy involving multiple chromosomes, while chromosomal microarray analysis (CMA) showed no abnormalities. Pregnancy termination was performed for fetus 1. WES identified compound heterozygous variants in BUB1B, i.e., c.2363_2364del (p.S788Cfs*29) and ss804270619: G>A, in both fetuses. Sanger sequencing confirmed paternal inheritance of c.2363_2364del and maternal inheritance of ss804270619:G>A. According to the American College of Medical Genetics and Genomics (ACMG) and Clinical Genome Resource (ClinGen) Standards and Guidelines for the Interpretation of Sequence Variants, the c.2363_2364del variant was classified as likely pathogenic (PVS1 + PM2_Supporting). Parental karyotyping demonstrated PCS traits, with a higher proportion of abnormal metaphases in the father.
The compound heterozygous variants c.2363_2364del (p.S788Cfs*29) and ss804270619: G>A in BUB1B may constitute the genetic etiology of the two MVA fetuses in this pedigree.
探究一个家系中两名患有嵌合型异倍体综合征(MVA)胎儿的遗传病因。
纳入一名于2023年11月16日就诊于山东省妇幼保健院医学遗传与产前诊断中心的30岁孕妇。收集该家系的临床资料,采集父母外周血样本及两名胎儿的羊水样本用于基因组DNA提取。对两名胎儿均进行全外显子测序(WES),随后进行Sanger测序以进行家系验证及候选变异的致病性分析。对父母进行染色体核型分析以量化早发性染色单体分离(PCS)的比例。本研究经山东省妇幼保健院医学伦理委员会批准(伦理编号:2024 - 034)。
两名胎儿在孕中期均表现出结构性脑异常和发育迟缓。羊水细胞染色体核型分析显示存在涉及多条染色体的低水平嵌合非整倍体,而染色体微阵列分析(CMA)未显示异常。对胎儿1实施了终止妊娠。WES在两名胎儿中均鉴定出BUB1B基因的复合杂合变异,即c.2363_2364del(p.S788Cfs*29)和ss804270619:G>A。Sanger测序证实c.2363_2364del为父系遗传,ss804270619:G>A为母系遗传。根据美国医学遗传学与基因组学学会(ACMG)和临床基因组资源(ClinGen)序列变异解读标准与指南,c.2363_2364del变异被分类为可能致病(PVS1 + PM2_Supporting)。父母染色体核型分析显示具有PCS特征,父亲中异常中期相的比例更高。
BUB1B基因中的复合杂合变异c.2363_2364del(p.S788Cfs*29)和ss804270619:G>A可能构成该家系中两名MVA胎儿的遗传病因。