Seitsonen Sanna, Helminen Merja, Jarva Hanna, Meri Seppo, Järvelä Irma
HUS:n silmäsairaala ja Helsingin yliopisto, Haartman-instituutti, lääketieteellisen genetiikan osasto 00251 Helsinki.
Duodecim. 2010;126(9):1071-5.
Properdin deficiency is a rare X-chromosomal single gene immunological disorder that causes an increased risk for severe infectious diseases, especially for Neisseria meningitidis in males. Here we describe a Finnish family with a novel mutation in the properdin gene. The index-patient was diagnosed to have meningococcal meningitis and severe properdin deficiency that was caused by a nonsense mutation in exon 9 (c.1164G > A; W377X). The mutation was inherited from his mother and was also detected in his brother, two maternal aunts, one female cousin and her son. Vaccinations and preventive antibiotics were given to all males at risk.
备解素缺乏症是一种罕见的X染色体单基因免疫疾病,会增加患严重传染病的风险,尤其是男性患脑膜炎奈瑟菌病的风险。在此,我们描述了一个芬兰家庭,其备解素基因存在一种新的突变。索引患者被诊断患有脑膜炎球菌性脑膜炎和严重的备解素缺乏症,这是由外显子9中的一个无义突变(c.1164G > A;W377X)引起的。该突变由他的母亲遗传而来,在他的兄弟、两位姨妈、一位表姐妹及其儿子中也检测到了该突变。对所有有风险的男性进行了疫苗接种和预防性抗生素治疗。