Ben Salah Ghada, Hadj Salem Ikhlas, Masmoudi Abderrahmen, Kallabi Fakhri, Turki Hamida, Fakhfakh Faiza, Ayadi Hamadi, Kamoun Hassen
Laboratory of Human Molecular Genetics, Faculty of Medicine, University of Sfax, Av. Majida Boulila, 3029, Sfax, Tunisia,
Mol Biol Rep. 2014 Nov;41(11):7373-80. doi: 10.1007/s11033-014-3624-5. Epub 2014 Aug 17.
The Bloom syndrome (BS) is an autosomic recessive disorder comprising a wide range of abnormalities, including stunted growth, immunodeficiency, sun sensitivity and increased frequency of various types of cancer. Bloom syndrome cells display a high level of genetic instability, including a 10-fold increase in the sister chromatid exchanges (SCE) level. Bloom syndrome arises through mutations in both alleles of the BLM gene, which was identified as a member of the RecQ helicase family. In this study, we screened a Tunisian family with three BS patients. Cytogenetic analysis showed several chromosomal aberrations, and an approximately 14-fold elevated SCE frequency in BS cells. A significant increase in SCE frequency was observed in some family members but not reaching the BS patients values, leading to suggest that this could be due to the heterozygous profile. Microsatellite genotyping using four fluorescent dye-labeled microsatellite markers revealed evidence of linkage to BLM locus and the healthy members, sharing higher SCE frequency, showed heterozygous haplotypes as expected. Additionally, the direct BLM gene sequencing identified a novel homozygous frameshift mutation c.3617-3619delAA (p.K1207fsX9) in BS patients and a heterozygous BLM mutation in the family members with higher SCE frequency. Our findings suggest that this latter mutation likely leads to a reduced BLM activity explaining the homologous recombination repair defect and, therefore, the increase in SCE. Based on the present data, the screening of this mutation could contribute to the rapid diagnosis of BS. The genetic confirmation of the mutation in BLM gene provides crucial information for genetic counseling and prenatal diagnosis.
布卢姆综合征(BS)是一种常染色体隐性疾病,表现出多种异常,包括生长发育迟缓、免疫缺陷、对阳光敏感以及各类癌症发病率增加。布卢姆综合征细胞表现出高度的遗传不稳定性,包括姐妹染色单体交换(SCE)水平增加10倍。布卢姆综合征是由BLM基因的两个等位基因发生突变引起的,该基因被鉴定为RecQ解旋酶家族的成员。在本研究中,我们对一个有三名BS患者的突尼斯家庭进行了筛查。细胞遗传学分析显示了几种染色体畸变,并且BS细胞中的SCE频率大约升高了14倍。在一些家庭成员中观察到SCE频率显著增加,但未达到BS患者的水平,这表明这可能是由于杂合子状态所致。使用四种荧光染料标记的微卫星标记进行微卫星基因分型,揭示了与BLM基因座连锁的证据,并且健康成员具有较高的SCE频率,如预期的那样显示出杂合单倍型。此外,直接对BLM基因进行测序,在BS患者中鉴定出一种新的纯合移码突变c.3617 - 3619delAA(p.K1207fsX9),在SCE频率较高的家庭成员中鉴定出杂合BLM突变。我们的研究结果表明,后一种突变可能导致BLM活性降低,从而解释了同源重组修复缺陷,进而导致SCE增加。基于目前的数据,对这种突变的筛查有助于BS的快速诊断。BLM基因突变的遗传学确认可为遗传咨询和产前诊断提供关键信息。