Clinical and Genetic Profiles of 11 Chinese Patients With Angelman Syndrome

11例中国安格曼综合征患者的临床和基因特征

阅读:3

Abstract

Angelman syndrome (AS) is a severe neurodevelopmental disorder resulting from different molecular mechanisms. Investigating the correlation between genotypes and phenotypes is crucial to facilitate accurate diagnosis and effective prevention strategies for this disorder. However, determining the genotypes of patients to analyze genotype‒phenotype correlations is challenging when parental genetic information is lacking. Therefore, we proposed a genotyping strategy for use with 11 unrelated Chinese patients with AS who were recruited for this study. The strategy involved a combination of methylation-specific polymerase chain reaction (MS-PCR), exome sequencing (ES), Sanger sequencing, and MS multiplexed ligation probe amplification (MS-MLPA). The results revealed that the number of molecular deletions involving the critical 15q11. 2-q13 region (54.5%) was lower than that reported in other studies of Chinese patients. In addition, the prevalence of patients with imprinting defects (IDs) (27.3%) and variants (18.2%) was greater, whereas the proportion of patients with uniparental disomy (UPDs) was lower. We also summarized the characteristics of patients with different genotypes and analyzed the correlations between genotypes and phenotypes. Compared with the consensus for diagnostic criteria, our results showed that several features were less common, including the combination of frequent laughing/smiling, tremulous limb movements, ataxia of gait, and microcephaly. Conversely, the incidence of both epilepsy and abnormal electroencephalograms (EEGs) was greater. Notably, a novel mutation in the UBE3A gene that had not been previously reported was identified in a family.

特别声明

1、本页面内容包含部分的内容是基于公开信息的合理引用;引用内容仅为补充信息,不代表本站立场。

2、若认为本页面引用内容涉及侵权,请及时与本站联系,我们将第一时间处理。

3、其他媒体/个人如需使用本页面原创内容,需注明“来源:[生知库]”并获得授权;使用引用内容的,需自行联系原作者获得许可。

4、投稿及合作请联系:info@biocloudy.com。