Yamada Mamiko, Suzuki Hisato, Miya Fuyuki, Takenouchi Toshiki, Kosaki Kenjiro
Center for Medical Genetics, Keio University School of Medicine, Tokyo, Japan.
Department of Pediatrics, Keio University School of Medicine, Tokyo, Japan.
Am J Med Genet A. 2022 Aug;188(8):2472-2478. doi: 10.1002/ajmg.a.62777. Epub 2022 May 14.
When a de novo balanced reciprocal translocation is identified in patients with multiple congenital abnormalities, attempts are often made to infer the relationship between the phenotype of the patient and genes in the proximity of the breakpoint. Here, we report a patient with intellectual disability, atrial septal defect, syndactyly, and cleft lip and palate who had an "apparently balanced" de novo reciprocal translocation t(4:18)(q31;q11.2) as well as a 7-Mb cryptic deletion spanning the HOXD cluster on chromosome 2q31 that was unrelated to the reciprocal translocation. Further analysis using a nanopore long-read sequencer showed complex rearrangements on both derivative chromosomes 4 and 18 and the deleted chromosome 2. First, the TLL1 locus, which is associated with atrial septal defect, was disrupted by the rearrangement involving chromosome 4. Second, the deleted interval at 2q31 included the entire HOXD cluster, the deletion of which is known to cause toe syndactyly, and the DLX1 and DLX2 loci, which are responsible for cleft lip and palate. Among the haplo-sensitive genes within the deleted interval on 2q31, only the RAPGEF4 gene is known to be associated with an autistic phenotype. Hence, most of the clinical features of the patient could be ascribed to specific genomic rearrangements. We have shown the effectiveness of long-read sequencing in defining, in detail, the likely effects of an apparently balanced translocation and cryptic deletion. The results of the present analysis suggest the possibility of phenotypic prediction through a detailed analysis of structural abnormalities, including balanced translocations and deletions.
当在患有多种先天性异常的患者中发现新发平衡相互易位时,人们常常试图推断患者的表型与断点附近基因之间的关系。在此,我们报告一名患有智力残疾、房间隔缺损、并指以及唇腭裂的患者,该患者有一个“看似平衡”的新发相互易位t(4:18)(q31;q11.2),以及一个跨越2号染色体q31上HOXD簇的7兆碱基隐匿性缺失,该缺失与相互易位无关。使用纳米孔长读测序仪进行的进一步分析显示,衍生染色体4和18以及缺失的2号染色体上均存在复杂重排。首先,与房间隔缺损相关的TLL1基因座被涉及染色体4的重排破坏。其次,2q31处的缺失区间包括整个HOXD簇,已知其缺失会导致脚趾并指,以及负责唇腭裂的DLX1和DLX2基因座。在2q31缺失区间内的单倍体敏感基因中,只有RAPGEF4基因已知与自闭症表型相关。因此,该患者的大多数临床特征可归因于特定的基因组重排。我们已经证明了长读测序在详细定义看似平衡的易位和隐匿性缺失可能产生的影响方面的有效性。本分析结果表明,通过对包括平衡易位和缺失在内的结构异常进行详细分析来预测表型是有可能的。