Kabuki Tomoyuki, Kawai Toshinao, Kin Yoshiaki, Joh Kohsuke, Ohashi Hirofumi, Kosho Tomoki, Yachie Akihiro, Kanegane Hirokazu, Miyawaki Toshio, Oh-ishi Tsutomu
Division of Infectious Diseases, Immunology and Allergy, Saitama Children's Medical Center.
Nihon Rinsho Meneki Gakkai Kaishi. 2003 Oct;26(5):299-303. doi: 10.2177/jsci.26.299.
A 2-month-old boy with a characteristic elfin face was diagnosed as having Williams syndrome by means of specific fluorescence in situ hybridization (FISH) analysis for a chromosomal microdeletion located in 7q11.23. He was suspected to have immunodeficiency because of a persistent enlargement of axillary lymphnodes after immunization with Bacille Calmette-Guerin (BCG) vaccine since 7 month-old of age. The nitroblue tetrazolium test (NBT) and the chemiluminescence test revealed an absence of superoxide production. Western blotting and DNA sequence analysis confirmed the diagnosis of p47-phox-deficient autosomal recessive chronic granulomatous disease (CGD) (A47 degrees CGD). The predominant genetic defect in A47 degrees CGD was a GT deletion at the beginning of exon 2 in neutrophil cytosol factor 1 gene (NCF1) located in 7q11.23. It suggests that CGD in this patient resulted from the hemizygosity of recessive genetic mutation in NCF1 located at 7q11.23 associated with Williams syndrome. In such a disease with the chromosomal microdeletion like Williams syndrome, we should consider a combination with autosomal recessive diseases, the genes of which are located in the hemizygous region.
一名2个月大、面部具有典型小精灵面容的男婴,通过针对位于7q11.23的染色体微缺失进行特异性荧光原位杂交(FISH)分析,被诊断为患有威廉姆斯综合征。自7月龄接种卡介苗(BCG)疫苗后,他的腋窝淋巴结持续肿大,因此怀疑存在免疫缺陷。硝基蓝四唑试验(NBT)和化学发光试验显示无超氧化物产生。蛋白质免疫印迹法和DNA序列分析确诊为p47-吞噬细胞氧化酶缺乏的常染色体隐性慢性肉芽肿病(CGD)(A47°CGD)。A47°CGD的主要基因缺陷是位于7q11.23的中性粒细胞胞质因子1基因(NCF1)外显子2起始处的GT缺失。这表明该患者的CGD是由位于7q11.23与威廉姆斯综合征相关的NCF1隐性基因突变的半合子状态导致的。在像威廉姆斯综合征这样存在染色体微缺失的疾病中,我们应考虑与常染色体隐性疾病合并,这些疾病的基因位于半合子区域。