Clinical Genetics Branch, Division of Cancer Epidemiology and Genetics, National Cancer Institute; NIH, Bethesda, MD, USA.
Laboratory of Molecular Gerontology, National Institute on Aging/National Institute of Health, Baltimore, MD, USA.
Br J Haematol. 2018 May;181(3):372-377. doi: 10.1111/bjh.15203.
In a previous whole exome sequencing of patients from 41 families with Hodgkin lymphoma, we identified two families with distinct heterozygous rare coding variants in POT1 (D224N and Y36H), both in a highly conserved region of the gene. POT1 D224N mutant did not bind to a single-stranded telomere oligonucleotide in vitro suggesting the mutation perturbs POT1's ability to bind to the telomeric G-rich overhang. Human HT1080 cells expressing POT1 D224N and lymphoblastoid cells carrying Y36H both showed increased telomere length and fragility in comparison to wild type cells. This strongly suggests that mutant POT1 causes chromosome instability and may play a role in lymphomagenesis in these families.
在先前对 41 个霍奇金淋巴瘤患者家族的全外显子组测序中,我们鉴定出两个家族存在 POT1 中两个不同的杂合稀有编码变异(D224N 和 Y36H),均位于该基因高度保守的区域。POT1 D224N 突变体在体外不能结合单链端粒寡核苷酸,提示该突变破坏了 POT1 结合端粒富含 G 的突出端的能力。与野生型细胞相比,表达 POT1 D224N 的人 HT1080 细胞和携带 Y36H 的淋巴母细胞显示出端粒长度增加和脆性增加。这强烈表明突变的 POT1 导致染色体不稳定性,并可能在这些家族的淋巴瘤发生中发挥作用。