The Centre for Applied Genomics and Program in Genetics and Genomic Biology, The Hospital for Sick Children, Toronto, Ontario M5G 1L7, Canada.
Nature. 2010 Jul 15;466(7304):368-72. doi: 10.1038/nature09146. Epub 2010 Jun 9.
The autism spectrum disorders (ASDs) are a group of conditions characterized by impairments in reciprocal social interaction and communication, and the presence of restricted and repetitive behaviours. Individuals with an ASD vary greatly in cognitive development, which can range from above average to intellectual disability. Although ASDs are known to be highly heritable ( approximately 90%), the underlying genetic determinants are still largely unknown. Here we analysed the genome-wide characteristics of rare (<1% frequency) copy number variation in ASD using dense genotyping arrays. When comparing 996 ASD individuals of European ancestry to 1,287 matched controls, cases were found to carry a higher global burden of rare, genic copy number variants (CNVs) (1.19 fold, P = 0.012), especially so for loci previously implicated in either ASD and/or intellectual disability (1.69 fold, P = 3.4 x 10(-4)). Among the CNVs there were numerous de novo and inherited events, sometimes in combination in a given family, implicating many novel ASD genes such as SHANK2, SYNGAP1, DLGAP2 and the X-linked DDX53-PTCHD1 locus. We also discovered an enrichment of CNVs disrupting functional gene sets involved in cellular proliferation, projection and motility, and GTPase/Ras signalling. Our results reveal many new genetic and functional targets in ASD that may lead to final connected pathways.
自闭症谱系障碍(ASD)是一组以相互社交互动和沟通障碍为特征的疾病,同时存在受限和重复行为。患有 ASD 的个体在认知发展方面差异很大,可从高于平均水平到智力残疾。尽管 ASD 被认为具有高度遗传性(约 90%),但其潜在的遗传决定因素仍在很大程度上未知。在这里,我们使用高密度基因分型阵列分析了 ASD 中罕见(<1%频率)拷贝数变异的全基因组特征。当将 996 名欧洲血统的 ASD 个体与 1287 名匹配对照进行比较时,发现病例携带更高的罕见基因拷贝数变异(CNV)全球负担(1.19 倍,P=0.012),尤其是在先前与 ASD 和/或智力障碍相关的基因座(1.69 倍,P=3.4×10(-4))。在 CNVs 中存在许多新生和遗传事件,有时在一个特定的家族中同时发生,这涉及到许多新的 ASD 基因,如 SHANK2、SYNGAP1、DLGAP2 和 X 连锁的 DDX53-PTCHD1 基因座。我们还发现了破坏涉及细胞增殖、投射和运动以及 GTPase/Ras 信号的功能基因集的 CNV 富集。我们的研究结果揭示了 ASD 中许多新的遗传和功能靶标,这些靶标可能导致最终的关联途径。
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