Mertz Line Granild Bie, Thaulov Per, Trillingsgaard Anegen, Christensen Rikke, Vogel Ida, Hertz Jens Michael, Ostergaard John R
Centre for Rare Diseases, Department of Pediatrics, Aarhus University Hospital, Denmark.
Psychiatric Hospital for Children and Adolescents, Aarhus University Hospital, Denmark.
Res Dev Disabil. 2014 Jul;35(7):1742-7. doi: 10.1016/j.ridd.2014.02.018. Epub 2014 Mar 19.
Angelman syndrome (AS) is a neurogenetic disorder characterized by intellectual disability, developmental delay, lack of speech, and epileptic seizures. Previous studies have indicated that children with AS due to 15q11.2-q13 deletions have a more severe developmental delay and present more often autistic features than those with AS caused by other genetic etiologies. The present study investigated the neurodevelopmental profiles of the different genetic etiologies of AS, and examined the evolution of mental development and autistic features over a 12-year period in children with a 15q11.2-q13 deletion. This study included 42 children with AS. Twelve had a Class I deletion, 18 had Class II deletions, three showed atypical large deletions, five had paternal uniparental disomy (pUPD) and four had UBE3A mutations. Children with a deletion (Class I and Class II) showed significantly reduced developmental age in terms of visual perception, receptive language, and expressive language when compared to those with a UBE3A mutation and pUPD. Within all subgroups, expressive language performance was significantly reduced when compared to the receptive performance. A follow-up study of seven AS cases with 15q11.2-q13 deletions revealed that over 12 years, the level of autistic features did not change, but both receptive and expressive language skills improved.
天使综合征(AS)是一种神经遗传性疾病,其特征为智力残疾、发育迟缓、语言缺失和癫痫发作。先前的研究表明,因15q11.2-q13缺失导致AS的儿童比由其他遗传病因引起AS的儿童发育迟缓更严重,且更常出现自闭症特征。本研究调查了AS不同遗传病因的神经发育概况,并在12年期间对15q11.2-q13缺失儿童的智力发育和自闭症特征演变进行了研究。本研究纳入了42例AS儿童。其中12例为I类缺失,18例为II类缺失,3例表现为非典型大片段缺失,5例为父源单亲二倍体(pUPD),4例为UBE3A突变。与UBE3A突变和pUPD儿童相比,缺失(I类和II类)儿童在视觉感知、接受性语言和表达性语言方面的发育年龄显著降低。在所有亚组中,与接受性表现相比,表达性语言表现显著降低。对7例15q11.2-q13缺失的AS病例进行的随访研究显示,在12年期间,自闭症特征水平没有变化,但接受性和表达性语言技能均有所提高。