Wielders Eva, Delzenne-Goette Elly, Dekker Rob, van der Valk Martin, Te Riele Hein
Division of Biological Stress Response, The Netherlands Cancer Institute/Antoni van Leeuwenhoek, Plesmanlaan 121, 1066 CX, Amsterdam, The Netherlands.
Fam Cancer. 2017 Apr;16(2):221-229. doi: 10.1007/s10689-016-9945-x.
Missense variants of DNA mismatch repair (MMR) genes pose a problem in clinical genetics as long as they cannot unambiguously be assigned as the cause of Lynch syndrome (LS). To study such variants of uncertain clinical significance, we have developed a functional assay based on direct measurement of MMR activity in mouse embryonic stem cells expressing mutant protein from the endogenous alleles. We have applied this protocol to a specific truncation mutant of MSH2 that removes 60 C-terminal amino acids and has been found in suspected LS families. We show that the stability of the MSH2/MSH6 heterodimer is severely perturbed, causing attenuated MMR in in vitro assays and cancer predisposition in mice. This mutation can therefore unambiguously be considered as deleterious and causative for LS.
只要DNA错配修复(MMR)基因的错义变异无法明确被认定为林奇综合征(LS)的病因,它们就在临床遗传学中构成一个问题。为了研究这类临床意义不确定的变异,我们开发了一种功能测定法,该方法基于直接测量在从内源性等位基因表达突变蛋白的小鼠胚胎干细胞中的MMR活性。我们已将此方案应用于MSH2的一种特定截短突变体,该突变体去除了60个C末端氨基酸,并且在疑似LS家族中被发现。我们表明,MSH2/MSH6异二聚体的稳定性受到严重干扰,导致体外测定中的MMR减弱以及小鼠患癌倾向增加。因此,这种突变可以明确被认为是有害的且是LS的病因。
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