Stalpers Xenia L, Spruijt Liesbeth, Yntema Helger G, Verrips Aad
Department of Pediatric Neurology, Canisius Wilhelmina Hospital, Nijmegen, The Netherlands.
J Child Neurol. 2012 Jan;27(1):90-3. doi: 10.1177/0883073811413832. Epub 2011 Jul 15.
Mutations in the X-linked cyclin dependent kinase like 5 (CDKL5) gene have been reported in approximately 80 patients since the first description in 2003. The clinical presentation partly corresponds with Rett syndrome, considering clinical features as intellectual disability, hypotonia, and poor visual, language, and motor development. However, these patients do not meet the consensus criteria for Rett syndrome since they lack the clear period of regression. Furthermore, in contrast to Rett syndrome, patients with CDKL5 mutations, have seizures or infantile spasms starting in the first weeks of life. We present clinical phenotype of 5 girls having a mutation in the CDKL5 gene. All mutations are novel and are pathogenic since they either lead to a frameshift in the reading frame or affect a consensus splice site. Four of the mutations are detected de novo in the affected girl.
自2003年首次报道以来,约80例患者被发现存在X连锁的类细胞周期蛋白依赖性激酶5(CDKL5)基因突变。考虑到智力残疾、肌张力减退以及视觉、语言和运动发育不良等临床特征,其临床表现部分与雷特综合征相符。然而,这些患者不符合雷特综合征的共识标准,因为他们没有明显的退化期。此外,与雷特综合征不同,CDKL5基因突变的患者在出生后的头几周就开始出现癫痫发作或婴儿痉挛症。我们报告了5名携带CDKL5基因突变女孩的临床表型。所有突变都是新发现的且具有致病性,因为它们要么导致阅读框移码,要么影响共有剪接位点。其中4种突变是在患病女孩中首次发现的。