Department of Clinical Sciences and Bioimages, 'G. d'Annunzio' University, Chieti-Pescara 66013, Italy.
Asian J Androl. 2010 Nov;12(6):819-26. doi: 10.1038/aja.2010.58. Epub 2010 Jul 26.
Congenital bilateral absence of vas deferens (CBAVD) is a manifestation of the mildest form of cystic fibrosis (CF) and is characterized by obstructive azoospermia in otherwise healthy patients. Owing to the availability of assisted reproductive technology, CBAVD patients can father children. These fathers are at risk of transmitting a mutated allele of the CF transmembrane conductance regulator (CFTR) gene, responsible for CF, to their offspring. The identification of mutations in both CFTR alleles in CBAVD patients is a crucial requirement for calculating the risk of producing a child with full-blown CF if the female partner is a healthy CF carrier. However, in the majority of CBAVD patients, conventional mutation screening is not able to detect mutations in both CFTR alleles, and this difficulty hampers the execution of correct genetic counselling. To obtain information about the most represented CFTR mutations in CBAVD patients, we analysed 23 CBAVD patients, 15 of whom had a single CFTR mutation after screening for 36 mutations and the 5T allele. The search for the second CFTR mutation in these cases was performed by using a triplex approach: (i) first, a reverse dot-blot analysis was performed to detect mutations with regional impact; (ii) next, multiple ligation-dependent probe amplification assays were conducted to search for large rearrangements; and (iii) finally, denaturing high-performance liquid chromatography was used to search for point mutations in the entire coding region. Using these approaches, the second CFTR mutation was detected in six patients, which increased the final detection rate to 60.8%.
先天性双侧输精管缺如(CBAVD)是囊性纤维化(CF)最轻微形式的表现,其特征是在其他方面健康的患者中出现梗阻性无精子症。由于辅助生殖技术的可用性,CBAVD 患者可以生育子女。这些父亲有将 CF 跨膜电导调节因子(CFTR)基因突变等位基因传给后代的风险,该基因负责 CF。CBAVD 患者中 CFTR 两个等位基因的突变鉴定是计算女性伴侣为健康 CF 携带者时产生完全 CF 患儿风险的关键要求。然而,在大多数 CBAVD 患者中,常规突变筛查无法检测到 CFTR 两个等位基因中的突变,这一困难阻碍了正确遗传咨询的实施。为了获得有关 CBAVD 患者中最常见 CFTR 突变的信息,我们分析了 23 名 CBAVD 患者,其中 15 名在筛选 36 种突变和 5T 等位基因后仅有一种 CFTR 突变。在这些情况下,通过三联体方法寻找第二个 CFTR 突变:(i)首先,进行反向斑点印迹分析以检测具有区域影响的突变;(ii)其次,进行多个连接依赖性探针扩增检测以寻找大的重排;(iii)最后,使用变性高效液相色谱法在整个编码区搜索点突变。使用这些方法,在 6 名患者中检测到第二个 CFTR 突变,最终检测率提高到 60.8%。