Fan-Huang Gordon, Schmidt Elizabeth L, Lee Moonsook, Sun Shixiang, Boull Christina, Maguiness Sheilagh M, Vijg Jan, Niedernhofer Laura J, Maslov Alexander Y
Department of Genetics, Albert Einstein College of Medicine, Bronx NY 10461, USA.
Institute on the Biology of Aging and Metabolism, University of Minnesota Medical School.
bioRxiv. 2025 Jul 19:2025.07.16.665113. doi: 10.1101/2025.07.16.665113.
We discovered a uniquely high spontaneous somatic mutational load in peripheral blood mononuclear cells (PBMCs) of Xeroderma Pigmentosum group C (XP-C) patients, characterized by elevated single nucleotide variants associated with mutation signatures SBS8 and SBS32, as well as an enrichment of single-nucleotide cytosine deletions. This hypermutability was markedly lower in fibroblasts, suggesting a replication-dependent mechanism of mutagenesis due to deficient global genome nucleotide excision repair (GG-NER). Our findings reveal distinct molecular subtypes within XP defined by spontaneous mutational load in normal blood cells and demonstrate that the exceptionally high mutation burden in XP-C leukemia originates from mutations already present prior to malignant transformation.
我们在着色性干皮病C组(XP-C)患者的外周血单个核细胞(PBMC)中发现了独特的高自发体细胞突变负荷,其特征是与突变特征SBS8和SBS32相关的单核苷酸变异升高,以及单核苷酸胞嘧啶缺失的富集。这种高突变性在成纤维细胞中明显较低,提示由于全球基因组核苷酸切除修复(GG-NER)缺陷导致的复制依赖性诱变机制。我们的研究结果揭示了XP中由正常血细胞中的自发突变负荷定义的不同分子亚型,并证明XP-C白血病中异常高的突变负担源于恶性转化之前就已存在的突变。