Huyghebaert Jolien, Christiaenssen Bregje, De Rademaeker Marjan, Van den Ende Jenneke, Vandeweyer Geert, Kooy R Frank, Mateiu Ligia, Annear Dale
Department of Medical Genetics, University of Antwerp, Antwerp, Belgium.
Department of Medical Genetics, University Hospital of Antwerp, Antwerp, Belgium.
Eur J Med Genet. 2025 Mar 7;75:105009. doi: 10.1016/j.ejmg.2025.105009.
In this study, we employed a multifaceted approach combining short-read whole genome sequencing (WGS) analyzed using Delly, cytogenomics using Bionano technology, and Sanger sequencing to identify the breakpoints of a balanced de novo paracentric inversion on chromosome 11, spanning approximately 64 Mb (inv11q13.3; q25). This inversion was discovered in a girl who presented with mild intellectual disability (ID), speech and language delays, a delay in motor development and attention deficit hyperactivity disorder (ADHD). Detailed analysis of the breakpoints revealed the disruption of two genes; SHANK2, which is critical for encoding a postsynaptic scaffolding protein at glutamatergic synapses in the brain, and LINC02714, a long non-coding RNA (lncRNA). Although SHANK2 is not listed in the OMIM database as a causative gene to this date, literature reports at least 21 cases where (likely) pathogenic variants in SHANK2 have been identified in patients with neurodevelopmental disorders (NDDs). A loss of function variant of the SHANK2 gene is in line with the clinical presentation of this patient. No additional genetic variants that could explain her phenotype were identified. In conclusion, by combining WGS, cytogenomics and Sanger sequencing techniques, we identified the exact breakpoints of a large inversion providing a likely molecular diagnosis for our patient.
在本研究中,我们采用了多方面的方法,将使用Delly分析的短读长全基因组测序(WGS)、使用Bionano技术的细胞基因组学以及桑格测序相结合,以确定11号染色体上一个平衡的新生臂内倒位的断点,该倒位跨度约64 Mb(inv11q13.3;q25)。此倒位在一名女孩中被发现,她表现出轻度智力障碍(ID)、言语和语言发育迟缓、运动发育迟缓以及注意力缺陷多动障碍(ADHD)。对断点的详细分析揭示了两个基因的破坏;SHANK2,它对于在大脑谷氨酸能突触处编码一种突触后支架蛋白至关重要,以及LINC02714,一种长链非编码RNA(lncRNA)。尽管截至目前SHANK2未在OMIM数据库中列为致病基因,但文献报道至少有21例在神经发育障碍(NDDs)患者中鉴定出(可能)SHANK2的致病变异。SHANK2基因的功能丧失变异与该患者的临床表现相符。未发现其他可解释其表型的遗传变异。总之,通过结合WGS、细胞基因组学和桑格测序技术,我们确定了一个大倒位的确切断点,为我们的患者提供了可能的分子诊断。