Wu Xingwu, Xu Qiang, Chen Ge, Huang Jialyv, Zhong Yanying, Tian Lifeng, Wu Qiongfang, Chen Jia
Reproductive Medicine Center, Jiangxi Maternal and Child Health Hospital, 318 Bayi Avenue, Nanchang, 330006, China.
Jiangxi Key Laboratory of Reproductive Health, Jiangxi Maternal and Child Health Hospital, Nanchang, China.
Sci Rep. 2025 Jan 21;15(1):2656. doi: 10.1038/s41598-025-87083-8.
To explore the genetic cause of a four-generation severe intellectual disability in a Chinese family using nanopore sequencing and to provide genetic counseling and reproductive guidance for family members. Multiple genetic analyses of the proband and family members were performed, including chromosome karyotype analysis, whole exome sequencing, nanopore sequencing, PCR amplification, and Sanger sequencing. The results of G-binding karyotyping, CGG repeats for FMR1, GGC repeats for NOTCH2NCL, and trio-whole-exome sequencing were negative for the proband and his parents. Nanopore sequencing showed that the proband carried 12q24.33 microduplication (3.26 Mb) and 22q13.33 microdeletion (1.5 Mb). According to the guidelines of the American Society for Medical Genetics and Genomics (ACMG), the 22q13.33 microdeletion was classified as pathogenic, whereas the 12q24.33 microduplication was classified as a variant of uncertain significance (VUS). The precise karyotype and location of chromosomal breakpoints in the patient and family members were determined through PCR. According to the results of Sanger sequencing, a cryptic balanced translocation was detected in the proband's father. Additionally, informative SNPs were identified near the breakpoints for preimplantation genetic testing for structure rearrangement (PGT-SR) treatment by nanopore sequencing. We identified a cryptic unbalanced translocation in a large Chinese family with Phelan-McDermid syndrome (22q13.33 deletion syndrome) by nanopore sequencing. Nanopore sequencing can be a powerful tool for the genetic diagnosis of unexplained intellectual disability and the detection of precise breakpoints of chromosomal rearrangement in PGT-SR treatment.
利用纳米孔测序技术探究一个中国家庭中四代人严重智力残疾的遗传病因,并为家庭成员提供遗传咨询和生育指导。对先证者及其家庭成员进行了多项基因分析,包括染色体核型分析、全外显子组测序、纳米孔测序、聚合酶链反应(PCR)扩增和桑格测序。先证者及其父母的G显带核型分析、FMR1基因的CGG重复序列、NOTCH2NCL基因的GGC重复序列以及三联体全外显子组测序结果均为阴性。纳米孔测序显示先证者携带12q24.33微重复(3.26 Mb)和22q13.33微缺失(1.5 Mb)。根据美国医学遗传学与基因组学学会(ACMG)的指南,22q13.33微缺失被分类为致病性变异,而12q24.33微重复被分类为意义未明的变异(VUS)。通过PCR确定了患者及其家庭成员染色体断点的精确核型和位置。根据桑格测序结果,在先证者父亲中检测到一个隐匿性平衡易位。此外,通过纳米孔测序在断点附近鉴定出信息性单核苷酸多态性(SNP),用于结构重排植入前基因检测(PGT-SR)治疗。我们通过纳米孔测序在一个患有费兰-麦克德米德综合征(22q13.33缺失综合征)的大型中国家庭中鉴定出一个隐匿性不平衡易位。纳米孔测序可以成为不明原因智力残疾基因诊断以及PGT-SR治疗中染色体重排精确断点检测的有力工具。