Lucas Morghan C, Keßler Thomas, Scharf Florentine, Steinke-Lange Verena, Klink Barbara, Laner Andreas, Holinski-Feder Elke
MGZ- Medical Genetics Center, Munich, Germany.
Medizinische Klinik und Poliklinik IV- Campus Innenstadt, Klinikum der Universität München, Munich, Germany.
Fam Cancer. 2025 May 3;24(2):42. doi: 10.1007/s10689-025-00470-y.
Interpreting variants of uncertain significance (VUS) in mismatch repair (MMR) genes remains a major challenge in managing Lynch syndrome and other hereditary cancer syndromes. This review outlines recommended VUS classification procedures, encompassing foundational and specialized methodologies tailored for MMR genes by expert organizations, including InSiGHT and ClinGen's Hereditary Colorectal Cancer/Polyposis Variant Curation Expert Panel (VCEP). Key approaches include: (1) functional data, encompassing direct assays measuring MMR proficiency such as in vitro MMR assays, deep mutational scanning, and MMR cell-based assays, as well as techniques like methylation-tolerant assays, proteomic-based approaches, and RNA sequencing, all of which provide critical functional evidence supporting variant pathogenicity; (2) computational data/tools, including in silico meta-predictors and models, which contribute to robust VUS classification when integrated with experimental evidence; and (3) enhanced variant detection to identify the actual causal variant through whole-genome sequencing and long-read sequencing to detect pathogenic variants missed by traditional methods. These strategies improve diagnostic precision, support clinical decision-making for Lynch syndrome, and establish a flexible framework that can be applied to other OMIM-listed genes.
解读错配修复(MMR)基因中意义未明的变异(VUS)仍然是管理林奇综合征和其他遗传性癌症综合征的一项重大挑战。本综述概述了推荐的VUS分类程序,包括由InSiGHT和ClinGen的遗传性结直肠癌/息肉病变异评估专家小组(VCEP)等专家组织针对MMR基因量身定制的基础和专门方法。关键方法包括:(1)功能数据,包括测量MMR功能的直接检测方法,如体外MMR检测、深度突变扫描和基于MMR细胞的检测,以及耐甲基化检测、基于蛋白质组学的方法和RNA测序等技术,所有这些都提供了支持变异致病性的关键功能证据;(2)计算数据/工具,包括计算机元预测器和模型,当与实验证据结合时,有助于进行可靠的VUS分类;(3)增强变异检测,通过全基因组测序和长读长测序来识别实际的致病变异,以检测传统方法遗漏的致病变异。这些策略提高了诊断的准确性,支持林奇综合征的临床决策,并建立了一个可应用于其他列入《在线人类孟德尔遗传》(OMIM)的基因的灵活框架。