State Key Laboratory of Complex Severe and Rare Diseases, Chinese Research Center for Behavior Medicine in Growth and Development, Department of Endocrinology, Peking Union Medical College Hospital, Chinese Academy of Medical Science and Peking Union Medical College, Beijing 100730, China.
Department of Obstetrics and Gynecology, National Clinical Research Center for Obstetric and Gynecologic Diseases, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College, Beijing 100730, China.
Genes (Basel). 2023 Apr 29;14(5):1016. doi: 10.3390/genes14051016.
(1) Background: Optical genome mapping (OGM) is a novel approach to identifying genomic structural variations with high accuracy and resolution. We report a proband with severe short stature caused by 46, XY, der (16) ins (16;15) (q23; q21.3q14) that was detected by OGM combined with other tests and review the clinical features of patients with duplication within 15q14q21.3; (2) Methods: OGM, whole exon sequencing (WES), copy number variation sequencing (CNV-seq), and karyotyping were used; (3) Results: The proband was a 10.7-year-old boy with a complaint of severe short stature (-3.41SDS) and abnormal gait. He had growth hormone deficiency, lumbar lordosis, and epiphyseal dysplasia of both femurs. WES and CNV-seq showed a 17.27 Mb duplication of chromosome 15, and there was an insertion in chromosome 16 found by karyotyping. Furthermore, OGM revealed that duplication of 15q14q21.3 was inversely inserted into 16q23.1, resulting in two fusion genes. A total of fourteen patients carried the duplication of 15q14q21.3, with thirteen previously reported and one from our center, 42.9% of which were de novo. In addition, neurologic symptoms (71.4%,10/14) were the most common phenotypes; (4) Conclusions: OGM combined with other genetic methods can reveal the genetic etiology of patients with the clinical syndrome, presenting great potential for use in properly diagnosing in the genetic cause of the clinical syndrome.
(1)背景:光学基因组图谱(OGM)是一种识别基因组结构变异的新方法,具有高精度和高分辨率。我们报道了一例由 46,XY,der(16)ins(16;15)(q23;q21.3q14)引起的严重身材矮小的先证者,该病例是通过 OGM 结合其他检测方法发现的,并回顾了 15q14q21.3 内重复的患者的临床特征;(2)方法:使用 OGM、全外显子测序(WES)、拷贝数变异测序(CNV-seq)和核型分析;(3)结果:先证者为 10.7 岁男孩,主诉严重身材矮小(-3.41SDS)和步态异常。他患有生长激素缺乏症、腰椎前凸和双侧股骨骨骺发育不良。WES 和 CNV-seq 显示染色体 15 存在 17.27Mb 的重复,核型分析发现染色体 16 有插入。此外,OGM 显示 15q14q21.3 的重复倒插入 16q23.1,导致两个融合基因。共有 14 例患者携带 15q14q21.3 的重复,其中 13 例为先前报道的,1 例为本中心的,42.9%为新生。此外,神经症状(71.4%,10/14)是最常见的表型;(4)结论:OGM 结合其他遗传方法可以揭示具有临床综合征的患者的遗传病因,为正确诊断临床综合征的遗传病因提供了巨大潜力。