Raun Nicholas, Mailo Janette, Spinelli Egidio, He Xu, McAvena Sarah, Brand Logan, O'Sullivan Julia, Andersen John, Richer Lawrence, Tang-Wai Richard, Bolduc Francois V
Division of Pediatric Neurology, University of Alberta, Edmonton, Alberta, Canada.
Division of Neurodevelopmental and Neuromotor Pediatrics, University of Alberta, Edmonton, Alberta, Canada.
Am J Med Genet A. 2017 Apr;173(4):972-977. doi: 10.1002/ajmg.a.38139.
As genome wide techniques become more common, an increasing proportion of patients with intellectual disability (ID) are found to have genetic defects allowing genotype-phenotype correlations. Previously, AKT3 deletion was suggested to be responsible for microcephaly in patients with 1q43-q44 deletion syndrome, but this does not correspond to all cases. We report a case of a de novo 1q44 deletion in an 8-year-old boy with microcephaly in whom AKT3 is not deleted. We used a systematic review of the literature, our patient, and network analysis to gain a better understanding of the genetic basis of microcephaly in 1q deletion patients. Our analysis showed that while AKT3 deletion is associated with more severe (≤3 SD) microcephaly in 1q43-q44 deletion patients, other genes may contribute to microcephaly in AKT3 intact patients with microcephaly and 1q43-44 deletion syndrome. We identified a potential role for HNRNPU, SMYD3, NLRP3, and KIF26B in microcephaly. Overall, our study highlights the need for network analysis and quantitative measures reporting in the phenotypic analysis of a complex genetic syndrome related to copy number variation.
随着全基因组技术越来越普遍,越来越多的智力障碍(ID)患者被发现存在基因缺陷,从而能够进行基因型-表型关联分析。此前,有人提出AKT3缺失是1q43-q44缺失综合征患者小头畸形的病因,但并非所有病例都如此。我们报告了一例8岁小头畸形男孩的新发1q44缺失病例,该病例中AKT3未缺失。我们通过对文献、我们的患者进行系统回顾以及网络分析,以更好地了解1q缺失患者小头畸形的遗传基础。我们的分析表明,虽然AKT3缺失与1q43-q44缺失患者中更严重(≤3标准差)的小头畸形相关,但其他基因可能在AKT3完整的小头畸形患者和1q43-44缺失综合征中导致小头畸形。我们确定了HNRNPU、SMYD3、NLRP3和KIF26B在小头畸形中的潜在作用。总体而言,我们的研究强调了在与拷贝数变异相关的复杂遗传综合征的表型分析中进行网络分析和定量测量报告的必要性。