Hunter New England Local Health District, Hunter Genetics, Cnr. Turton & Tinonee Rds, Waratah, NSW, AUS 2298, Australia; University of Sydney, Faculty of Medicine and Health, Edward Ford Building (A27), Fisher Road, Camperdown, NSW, AUS 2006, Australia.
Hunter New England Local Health District, Hunter Genetics, Cnr. Turton & Tinonee Rds, Waratah, NSW, AUS 2298, Australia; University of Newcastle, Callaghan, NSW, AUS 2308, Australia.
Eur J Med Genet. 2020 Dec;63(12):104092. doi: 10.1016/j.ejmg.2020.104092. Epub 2020 Oct 28.
The Nuclear Factor I (NFI) transcription family (NFIA, NFIB and NFIX) have been implicated in a range of developmental pathologies, including corpus callosum, craniofacial, urinary tract abnormalities, as well in the development of a number of neurodevelopmental developmental phenotypes including muscular hypotonia, motor and speech delay, attention deficit disorder, autism spectrum disorder, and behavioural abnormalities. NFIB haploinsufficiency has only recently been presented as a cause for macrocephaly-intellectual disability syndrome, with comparable phenotypes to NFIA related disorder. We add another patient with a previously reported nonsense variant in the NFIB who has Autism Spectrum Disorder level 2, agenesis of the corpus callosum, ADHD, obsessive compulsive Disorder and an intellectual disability. A clinical exome analysis identified a nonsense variant, c.265C > T, p.(Arg89*) involving exon 2 of NFIB (ClinVar variation ID: 424,344). A brain MRI demonstrated agenesis of the corpus callosum.
核因子 I(NFI)转录家族(NFIA、NFIB 和 NFIX)与多种发育病理学有关,包括胼胝体、颅面、泌尿道异常,以及许多神经发育发育表型的发展,包括肌肉张力减退、运动和言语延迟、注意力缺陷障碍、自闭症谱系障碍和行为异常。NFIB 单倍不足最近才被提出作为巨脑-智力障碍综合征的原因,与 NFIA 相关疾病具有可比的表型。我们添加了另一位患者,该患者在 NFIB 中存在先前报道的无意义变异,患有自闭症谱系障碍 2 级、胼胝体发育不全、ADHD、强迫症和智力障碍。临床外显子组分析确定了一个无意义变异 c.265C>T,p.(Arg89*),涉及 NFIB 的外显子 2(ClinVar 变异 ID:424,344)。脑部 MRI 显示胼胝体发育不全。