Department of Laboratory Medicine and Pathology, Mayo Clinic, Rochester, Minnesota.
Hum Mutat. 2014 Feb;35(2):151-64. doi: 10.1002/humu.22478. Epub 2013 Dec 3.
Missense variants in the BRCA2 gene are routinely detected during clinical screening for pathogenic mutations in patients with a family history of breast and ovarian cancer. These subtle changes frequently remain of unknown clinical significance because of the lack of genetic information that may help establish a direct correlation with cancer predisposition. Therefore, alternative ways of predicting the pathogenicity of these variants are urgently needed. Since BRCA2 is a protein involved in important cellular mechanisms such as DNA repair, replication, and cell cycle control, functional assays have been developed that exploit these cellular activities to explore the impact of the variants on protein function. In this review, we summarize assays developed and currently utilized for studying missense variants in BRCA2. We specifically depict details of each assay, including variants of uncertain significance analyzed, and describe a validation set of (genetically) proven pathogenic and neutral missense variants to serve as a golden standard for the validation of each assay. Guidelines are proposed to enable implementation of laboratory-based methods to assess the impact of the variant on cancer risk.
在有乳腺癌和卵巢癌家族史的患者中,进行致病性突变的临床筛查时,通常会检测到 BRCA2 基因中的错义变异。由于缺乏可能有助于确定与癌症易感性直接相关性的遗传信息,这些细微的变化经常仍然具有未知的临床意义。因此,迫切需要其他预测这些变异致病性的方法。由于 BRCA2 是一种参与重要细胞机制的蛋白质,如 DNA 修复、复制和细胞周期控制,因此已经开发了利用这些细胞活性来探索变异对蛋白质功能影响的功能测定法。在这篇综述中,我们总结了用于研究 BRCA2 中错义变异的开发和当前使用的测定法。我们特别描述了每个测定法的细节,包括分析的意义不明的变异,并描述了一组(遗传)已证实的致病性和中性错义变异作为每个测定法验证的金标准。提出了指导方针,以能够实施基于实验室的方法来评估变异对癌症风险的影响。