Al-Amri Ahmed, Saegh Abeer Al, Al-Mamari Watfa, El-Asrag Mohammed E, Ivorra Jose L, Cardno Alastair G, Inglehearn Chris F, Clapcote Steven J, Ali Manir
Section of Ophthalmology and Neuroscience, Leeds Institute of Biomedical and Clinical Sciences, University of Leeds, United Kingdom.
School of Biomedical Sciences, University of Leeds, United Kingdom.
Am J Med Genet A. 2016 Jul;170(7):1826-31. doi: 10.1002/ajmg.a.37690. Epub 2016 May 5.
Intellectual disability (ID) is the term used to describe a diverse group of neurological conditions with congenital or juvenile onset, characterized by an IQ score of less than 70 and difficulties associated with limitations in cognitive function and adaptive behavior. The condition can be inherited or caused by environmental factors. The genetic forms are heterogeneous, with mutations in over 500 known genes shown to cause the disorder. We report a consanguineous Omani family in which multiple individuals have ID and developmental delay together with some variably present features including short stature, microcephaly, moderate facial dysmorphism, and congenital malformations of the toes or hands. Homozygosity mapping combined with whole exome next generation sequencing identified a novel homozygous single base pair deletion in TUSC3, c.222delA, p.R74 fs. The mutation segregates with the disease phenotype in a recessive manner and is absent in 60,706 unrelated individuals from various disease-specific and population genetic studies. TUSC3 mutations have been previously identified as causing either syndromic or non-syndromic ID in patients from France, Italy, Iran and Pakistan. This paper supports the previous clinical descriptions of the condition caused by TUSC3 mutations and describes the seventh family with mutations in this gene, thus contributing to the genetic spectrum of mutations. This is the first report of a family from the Arabian peninsula with this form of ID. © 2016 Wiley Periodicals, Inc.
智力残疾(ID)是一个术语,用于描述一组具有先天性或幼年起病的多种神经疾病,其特征为智商得分低于70,以及与认知功能和适应性行为受限相关的困难。这种情况可以是遗传的,也可由环境因素引起。遗传形式具有异质性,已发现500多个已知基因的突变可导致该疾病。我们报告了一个阿曼近亲家庭,其中多个个体患有智力残疾和发育迟缓,同时伴有一些可变出现的特征,包括身材矮小、小头畸形、中度面部畸形以及脚趾或手部先天性畸形。纯合子定位结合全外显子组下一代测序在TUSC3基因中鉴定出一个新的纯合单碱基对缺失,即c.222delA,p.R74 fs。该突变以隐性方式与疾病表型共分离,在来自各种疾病特异性和群体遗传学研究的60,706名无关个体中不存在。先前已确定TUSC3突变在来自法国、意大利、伊朗和巴基斯坦的患者中可导致综合征性或非综合征性智力残疾。本文支持先前对由TUSC3突变引起的疾病的临床描述,并描述了该基因发生突变的第七个家庭,从而丰富了突变的遗传谱。这是来自阿拉伯半岛的一个家庭患这种形式智力残疾的首次报告。© 2016威利期刊公司