U.O. Pediatria e Terapia Intensiva Neonatale, Dipartimento Materno Infantile, Università degli Studi di Palermo, Palermo, Italy.
Eur J Pediatr. 2010 Apr;169(4):415-9. doi: 10.1007/s00431-009-1037-6. Epub 2009 Aug 16.
X-linked hydrocephalus is due to mutations in the L1 neuronal cell adhesion molecule (L1CAM) gene. L1 protein plays a key role in neurite outgrowth, axonal guidance, and pathfinding during the development of the nervous system. We report on a familial case diagnosed by prenatal ultrasonographic examination, with cerebellar hypoplasia, agenesis of the corpus callosum, and the bilateral overlapping of the second and third fingers of the hand. Sequencing of the L1CAM gene showed a novel missense mutation in exon 14: transition of a guanine to cytosine at position 1777 (c.1777G>C), which led to an amino acid change of alanine to proline at position 593 (Ala593Pro) in the sixth immunoglobulin domain of the L1 protein. The L1CAM mutation testing should be considered in fetuses with ultrasonographic signs of hydrocephalus and a positive family history compatible with X-linked inheritance. We agree with previous reports that suggest also considering limb abnormalities other than adducted thumbs in addition to classical neurological disgenesis, as characteristic for L1-disease.
X 连锁脑积水是由于 L1 神经元细胞黏附分子(L1CAM)基因的突变引起的。L1 蛋白在神经系统发育过程中,对于神经突生长、轴突导向和寻路起着关键作用。我们报告了一个通过产前超声检查诊断的家族性病例,其表现为小脑发育不全、胼胝体发育不全以及手的第二和第三指双侧重叠。对 L1CAM 基因进行测序显示,在第 14 外显子中存在一个新的错义突变:1777 位的鸟嘌呤到胞嘧啶的转换(c.1777G>C),导致 L1 蛋白第六免疫球蛋白结构域第 593 位的丙氨酸到脯氨酸的氨基酸变化(Ala593Pro)。当胎儿存在超声脑积水征象且家族史符合 X 连锁遗传时,应考虑进行 L1CAM 基因突变检测。我们同意之前的报告,即建议除了经典的神经发育不全外,还应考虑除内收拇指以外的其他肢体异常,这是 L1 疾病的特征。