Zhang Xuan, Lu Hongjuan, Yang Hanran, Ji Yichen, Liu Huixin, Liu Wenjian, Li Jiayi, Yang Zhixian, Sun Wei
Department of Neurology, Xuanwu Hospital, Capital Medical University, Beijing, China.
Department of Pediatrics, Peking University First Hospital, Beijing, China.
Front Genet. 2023 Jun 14;14:1174314. doi: 10.3389/fgene.2023.1174314. eCollection 2023.
Structural rearrangements of chromosome 4p gives rise to a group of rare genomic disorders that mainly result in two different clinical entities: Wolf-Hirschhorn syndrome (WHS) and partial 4p trisomy. The severity of the phenotype depends on the size of the deletion or locus duplication. Here, we present two unrelated individuals with a copy number variation of chromosome 4p. Inverted duplication deletions (inv dup-del) in 4p are particularly rare. Case 1 describes a 15-year-old girl with a 1.055 Mb deletion of terminal 4p, distal to the recognized critical region of WHS, and a large duplication of 9.6 Mb in size from 4p16.3 to p16.1. She had postnatal development delay, intellectual disability (especially pronounced in speech), seizure/electroencephalogram anomalies, and facial dysmorphic features. This unusual chromosomal imbalance resulted in the WHS phenotype rather than the 4p trisomy syndrome phenotype. Case 2 describes a 21-month-old boy with a 1.386 Mb terminal 4p deletion who presented with slight developmental delay, borderline intellectual disability, and seizures. Combined with previous reported cases of 4 pter del-dup or pure 4p terminal deletions, our observations suggest that terminal chromosome 4p deletion is more pathogenic than the concomitant partial 4p duplication, and some regions of the 4p terminal may have regulatory effects on the remaining region of 4p. About nine cases have been reported thus far to date, and our study delineates further genotype-phenotype correlations about terminal 4p duplication-deletions for predicting disease prognosis and patient counseling.
4号染色体短臂的结构重排会引发一组罕见的基因组疾病,主要导致两种不同的临床病症:沃尔夫-赫希霍恩综合征(WHS)和部分4号染色体短臂三体。表型的严重程度取决于缺失或基因座重复的大小。在此,我们报告了两名具有4号染色体短臂拷贝数变异的无关个体。4号染色体短臂的倒位重复缺失(inv dup-del)尤为罕见。病例1描述了一名15岁女孩,其4号染色体短臂末端有一个1.055 Mb的缺失,位于公认的WHS关键区域远端,还有一个从4p16.3到p16.1的9.6 Mb的大重复。她有产后发育延迟、智力残疾(尤其是语言方面明显)、癫痫/脑电图异常以及面部畸形特征。这种不寻常的染色体失衡导致了WHS表型而非4号染色体短臂三体综合征表型。病例2描述了一名21个月大的男孩,有一个1.386 Mb的4号染色体短臂末端缺失,表现为轻度发育延迟、边缘智力残疾和癫痫发作。结合先前报道的4号染色体短臂末端缺失-重复或单纯4号染色体短臂末端缺失的病例,我们的观察结果表明,4号染色体短臂末端缺失比伴随的部分4号染色体短臂重复更具致病性,并且4号染色体短臂末端的某些区域可能对4号染色体短臂的其余区域具有调控作用。迄今为止,大约已报道了9例,我们的研究进一步阐明了4号染色体短臂末端重复-缺失的基因型-表型相关性,以预测疾病预后并为患者提供咨询。