Department of Laboratory Medicine and Genetics, Samsung Medical Center, Sungkyunkwan University School of Medicine, Seoul, Korea.
J Korean Med Sci. 2010 Oct;25(10):1513-7. doi: 10.3346/jkms.2010.25.10.1513. Epub 2010 Sep 20.
Incontinentia pigmenti (IP) is a rare X-linked dominant disorder characterized by highly variable abnormalities of the skin, eyes and central nervous system. A mutation of the nuclear factor-κB essential modulator (NEMO) located at Xq28 is believed to play a role in pathogenesis and the mutation occurs mostly in female patients due to fatal consequence of the mutation in males in utero. This study was designed to identify the common NEMO rearrangement in four Korean patients with IP. Deletion of exons 4 to 10 in the NEMO, the most common mutation in IP patients, was detected in all of the patients by the use of long-range PCR analysis. This method enabled us to discriminate between NEMO and pseudogene rearrangements. Furthermore, all of the patients showed skewed XCI patterns, indicating pathogenicity of IP was due to cells carrying the mutant X chromosome. This is the first report of genetically confirmed cases of IP in Korea.
遗传性皮肤病(IP)是一种罕见的 X 连锁显性遗传疾病,其特征是皮肤、眼睛和中枢神经系统高度异常。位于 Xq28 的核因子-κB 必需调节剂(NEMO)的突变被认为在发病机制中起作用,由于突变在男性胎儿中会导致致命后果,因此突变主要发生在女性患者中。本研究旨在鉴定四名韩国遗传性皮肤病患者中常见的 NEMO 重排。通过长距离 PCR 分析,在所有患者中均检测到 NEMO 中最常见的突变缺失外显子 4 至 10。该方法能够区分 NEMO 和假基因重排。此外,所有患者均显示出偏性 XCI 模式,表明 IP 的致病性是由于携带突变 X 染色体的细胞所致。这是韩国首例遗传性皮肤病的基因确诊病例报告。