Prontera Paolo, Ottaviani Valentina, Toccaceli Daniela, Rogaia Daniela, Ardisia Carmen, Romani Rita, Stangoni Gabriela, Pierini Angiolo, Donti Emilio
Centro di Riferimento Regionale di Genetica Medica, Azienda Ospedaliera-Universitaria di Perugia, Perugia, Italy.
Am J Med Genet A. 2014 Dec;164A(12):3137-41. doi: 10.1002/ajmg.a.36741. Epub 2014 Sep 24.
The most frequent causes of Intellectual Disability (ID)/Autism Spectrum Disorders (ASDs) are chromosomal abnormalities, genomic rearrangements and submicroscopic deletions coupled with duplications. We report here on an 11-year-old girl showing autism, macrocephaly, and facial dysmorphism, in which array-CGH showed a de novo microdeletion of ∼114 Kb in the 14q11.2 chromosomal region, involving the SUPT16H, CHD8, and RAB2B genes. Four patients with ID and/or ASD and/or macrocephaly with overlapping deletions have been previously described: three showed very large rearrangements (>1 Mb), while one had a microdeletion of ∼101 Kb, largely overlapping the one reported herein. The minimal critical region, considering present and previous cases, contains the SUPT16H and CHD8 genes. Notably, recent studies also disclosed CHD8 heterozygous loss-of-function mutations in patients with ASD and macrocephaly. Our finding shows the presence of a recurrent microdeletion associated with a clinically recognizable phenotype, and further on underlines the pivotal role of CHD8 gene in the pathogenesis of the disorder.
智力残疾(ID)/自闭症谱系障碍(ASD)最常见的病因是染色体异常、基因组重排以及亚显微水平的缺失和重复。我们在此报告一名11岁女孩,她患有自闭症、巨头畸形和面部畸形,阵列比较基因组杂交(array-CGH)显示其14q11.2染色体区域存在一个约114 kb的新生微缺失,涉及SUPT16H、CHD8和RAB2B基因。此前已描述了4例患有ID和/或ASD和/或巨头畸形且存在重叠缺失的患者:3例显示有非常大的重排(>1 Mb),而1例有一个约101 kb的微缺失,与本文报道的微缺失大部分重叠。综合目前和既往病例来看,最小关键区域包含SUPT16H和CHD8基因。值得注意的是,最近的研究还揭示了患有ASD和巨头畸形的患者中存在CHD8杂合功能丧失突变。我们的发现表明存在一种与临床可识别表型相关的复发性微缺失,并且进一步强调了CHD8基因在该疾病发病机制中的关键作用。