Casals T, De-Gracia J, Gallego M, Dorca J, Rodríguez-Sanchón B, Ramos M D, Giménez J, Cisteró-Bahima A, Olveira C, Estivill X
Medical and Molecular Genetics Center, Institut Recerca Oncològica (IRO), Hospital Duran i Reynals, Gran Via s/n km 2.7, 09807 Barcelona, Spain.
Clin Genet. 2004 Jun;65(6):490-5. doi: 10.1111/j.0009-9163.2004.00265.x.
While all patients with cystic fibrosis (CF) have mutations in both CFTR alleles, often only one CFTR change is detected in patients with other lung disorders. The aim of this study was to investigate whether heterozygosity for CFTR mutations could be a determinant risk factor in the development of bronchiectasis in adult patients. We have performed the CFTR gene analysis in a cohort of 55 bronchiectasis adult patients with unknown etiology. The 5T variant (TG)m and the M470V polymorphisms were also analyzed. A general population in which the same molecular analysis was previously performed was used as the control group. The mutational spectrum of patients was also compared with that found in our CF population. CFTR mutations/variants were found in 20 patients (36%), 14 with only one mutant gene (25%). All six patients colonized by Staphylococcus aureus presented with at least one CFTR change (p = 0.001). No statistical significance was observed between patients with and without mutations for other clinical features. The 5T variant was found in four patients. Additionally, 90% of patients with mutations had the more functional M470 allele (p < 0.001). These results suggest the involvement of the CFTR gene in bronchiectasis of unknown etiology in adult patients.
虽然所有囊性纤维化(CF)患者的CFTR等位基因均有突变,但在患有其他肺部疾病的患者中,通常仅检测到一个CFTR变化。本研究的目的是调查CFTR突变的杂合性是否可能是成年患者支气管扩张发生的决定性危险因素。我们对55例病因不明的成年支气管扩张患者进行了CFTR基因分析。还分析了5T变异体(TG)m和M470V多态性。将先前进行相同分子分析的普通人群用作对照组。还将患者的突变谱与我们CF患者群体中的突变谱进行了比较。在20例患者(36%)中发现了CFTR突变/变异体,其中14例仅有一个突变基因(25%)。所有6例金黄色葡萄球菌定植患者均至少有一个CFTR变化(p = 0.001)。对于其他临床特征,有突变和无突变的患者之间未观察到统计学意义。在4例患者中发现了5T变异体。此外,90%的突变患者具有功能更强的M470等位基因(p < 0.001)。这些结果表明CFTR基因参与了成年患者病因不明的支气管扩张。