Ververi Athina, Islam Lily, Bewes Beverley, Busby Louise, Sullivan Caroline, Canham Natalie
North West Thames Regional Genetics Service, London, UK.
Cytogenet Genome Res. 2017;152(3):132-136. doi: 10.1159/000480030. Epub 2017 Sep 13.
Angelman syndrome (AS) is characterised by developmental delay, lack of speech, seizures, a characteristic behavioural profile with a happy demeanour, microcephaly, and ataxia. More than two-thirds of cases are due to an approximately 5-Mb interstitial deletion of the imprinted region 15q11.2q13, which is usually de novo. The rest are associated with point mutations in the UBE3A gene, imprinting defects, and paternal uniparental disomy. Small intragenic UBE3A deletions have rarely been described. They are usually maternally inherited, increasing the recurrence risk to 50%, and may be missed by conventional testing (methylation studies and UBE3A gene sequencing). We describe a boy with AS due to an 11.7-kb intragenic deletion. The deletion was identified by array-CGH and was subsequently detected in his affected first cousin and unaffected maternal grandfather, mother, and aunt, confirming the silencing of the paternal allele. The patient had developmental delay, speech impairment, a happy demeanour, microcephaly, and an abnormal EEG, but no seizures by the age of 4 years. Delineation of the underlying genetic mechanism is of utmost importance for reasons of genetic counselling, as well as appropriate management and prognosis. Alternative techniques, such as array-CGH and MLPA, are necessary when conventional testing for AS has failed to identify the underlying genetic mechanism.
天使综合征(AS)的特征为发育迟缓、言语缺失、癫痫发作、具有快乐神情的特征性行为表现、小头畸形和共济失调。超过三分之二的病例是由于15q11.2q13印记区域约5 Mb的间质性缺失,这种缺失通常是新发的。其余病例与UBE3A基因突变、印记缺陷和父源单亲二体有关。很少有关于UBE3A基因小的基因内缺失的描述。它们通常是母系遗传,使复发风险增加到50%,并且可能会被传统检测(甲基化研究和UBE3A基因测序)遗漏。我们描述了一名因11.7 kb基因内缺失而患有天使综合征的男孩。该缺失通过阵列比较基因组杂交(array-CGH)得以鉴定,随后在其患病的一级表亲和未患病的外祖父、母亲及姨妈中检测到,证实了父源等位基因的沉默。该患者有发育迟缓、言语障碍、快乐神情、小头畸形以及脑电图异常,但4岁时未出现癫痫发作。明确潜在的遗传机制对于遗传咨询以及恰当的管理和预后至关重要。当对天使综合征的传统检测未能识别潜在遗传机制时,诸如阵列比较基因组杂交和多重连接探针扩增(MLPA)等替代技术是必要的。