Morak Monika, Käsbauer Sarah, Kerscher Martina, Laner Andreas, Nissen Anke M, Benet-Pagès Anna, Schackert Hans K, Keller Gisela, Massdorf Trisari, Holinski-Feder Elke
Medizinische Klinik und Poliklinik IV, Campus Innenstadt, Klinikum der Universität München, Ziemssenstr. 1, 80336, Munich, Germany.
MGZ - Center of Medical Genetics, Bayerstr. 3-5, 80335, Munich, Germany.
Fam Cancer. 2017 Oct;16(4):491-500. doi: 10.1007/s10689-017-9975-z.
Lynch Syndrome (LS) is the most common dominantly inherited colorectal cancer (CRC) predisposition and is caused by a heterozygous germline defect in one of the DNA mismatch repair (MMR) genes MLH1, MSH2, MSH6, or PMS2. High microsatellite instability (MSI-H) and loss of MMR protein expression in tumours reflecting a defective MMR are indicators for LS, as well as a positive family history of early onset CRC. MSH2 and MSH6 form a major functional heterodimer, and MSH3 is an alternative binding partner for MSH2. So far, the role of germline MSH3 variants remains unclear, as to our knowledge heterozygous truncating variants are not regarded causative for LS, but were detected in patients with CRC, and recently biallelic MSH3 defects have been identified in two patients with adenomatous polyposis. By gene screening we investigated the role of MSH3 in 11 LS patients with truncating MSH6 germline variants and an unexplained MSH2 protein loss in their corresponding MSI-H tumours. We report the first two LS patients harbouring heterozygous germline variants c.1035del and c.2732T>G in MSH3 coincidentally with truncating variants in MSH6. In the patient with truncating germline variants in MSH3 and MSH6, two additional somatic second hits in both genes abrogate all binding partners for the MSH2 protein which might subsequently be degraded. The clinical relevance of MSH3 germline variants is currently under re-evaluation, and heterozygous MSH3 defects alone do not seem to induce a LS phenotype, but might aggravate the MSH6 phenotype in affected family members.
林奇综合征(LS)是最常见的显性遗传性结直肠癌(CRC)易患因素,由DNA错配修复(MMR)基因MLH1、MSH2、MSH6或PMS2中的一个杂合种系缺陷引起。肿瘤中的高微卫星不稳定性(MSI-H)和反映MMR缺陷的MMR蛋白表达缺失是LS的指标,以及早发性CRC的阳性家族史。MSH2和MSH6形成主要的功能性异二聚体,MSH3是MSH2的替代结合伙伴。到目前为止,种系MSH3变异的作用仍不清楚,据我们所知,杂合性截短变异不被认为是LS的病因,但在CRC患者中被检测到,最近在两名腺瘤性息肉病患者中发现了双等位基因MSH3缺陷。通过基因筛查,我们研究了MSH3在11例具有截短MSH6种系变异且其相应MSI-H肿瘤中MSH2蛋白缺失原因不明的LS患者中的作用。我们报告了首例两名LS患者,其MSH3中携带杂合种系变异c.1035del和c.2732T>G,同时伴有MSH6中的截短变异。在MSH3和MSH6中具有截短种系变异的患者中,两个基因中另外两个体细胞二次打击消除了MSH2蛋白的所有结合伙伴,这些伙伴随后可能被降解。MSH3种系变异的临床相关性目前正在重新评估中,单独的杂合性MSH3缺陷似乎不会诱导LS表型,但可能会加重受影响家庭成员的MSH6表型。