Laboratorio de Citogenomica, Departamento de Patologia, Faculdade de Medicina FMUSP, Universidade de Sao Paulo, Sao Paulo, SP, Brazil.
Unidade de Genetica, Departamento de Pediatria, Instituto da Crianca, Hospital das Clinicas HCFMUSP, Faculdade de Medicina, Universidade de Sao Paulo, Sao Paulo, SP, Brazil.
Mol Genet Genomic Med. 2020 Apr;8(4):e1133. doi: 10.1002/mgg3.1133. Epub 2020 Feb 19.
Bloom syndrome (BS) is a rare autosomal recessive chromosome instability disorder. The main clinical manifestations are growth deficiency, telangiectasic facial erythema, immunodeficiency, and increased risk to develop neoplasias at early age. Cytogenetic test for sister chromatid exchanges (SCEs) is used as a diagnostic marker for BS. In addition, most patients also present mutations in the BLM gene, related to defects in the DNA repair mechanism. However, the molecular mechanism behind the pathogenicity of BS is still not completely understood.
We describe two patients confirmed with BS by SCE and molecular analysis. Also, we performed the gene expression profile by the RNA-seq methodology in mRNA transcripts for differential gene expression analysis using as a biological condition for comparison BS versus health controls.
We detected 216 differentially expressed genes related to immunological pathways such as positive regulation and activation of B cells, immune effector process and absence of difference of DNA repair genes expression. In addition; we also observed differentially expressed genes associated with apoptosis control, such as BCL2L1, CASP7, CDKN1A, E2F2, ITPR, CD274, TNFAIP6, TNFRSF25, TNFRSF13C, and TNFRSF17.
Our results suggest that the combination of altered expression of genes involved in signaling pathways of immune response and apoptosis control may contribute directly to the main characteristics observed in BS, such as recurrent infections, growth failure, and high risk of cancer. Transcriptome studies of other instability syndromes could allow a more accurate analysis of the relevant gene interactions associated with the destabilization of the genome. This is a first description of the profile of differential gene expression related to immunological aspects detected in patients with BS by RNA-seq.
布卢姆综合征(BS)是一种罕见的常染色体隐性染色体不稳定疾病。主要临床表现为生长发育迟缓、毛细血管扩张性面部红斑、免疫缺陷以及在早年增加罹患肿瘤的风险。姐妹染色单体交换(SCE)的细胞遗传学测试被用作 BS 的诊断标志物。此外,大多数患者还存在 BLM 基因的突变,与 DNA 修复机制缺陷有关。然而,BS 的发病机制背后的分子机制仍不完全清楚。
我们描述了通过 SCE 和分子分析确诊的 2 例 BS 患者。此外,我们通过 RNA-seq 方法进行了基因表达谱分析,以比较 BS 与健康对照的差异基因表达分析。
我们检测到 216 个差异表达基因,这些基因与免疫途径相关,如 B 细胞的正调控和激活、免疫效应子过程和 DNA 修复基因表达的无差异。此外,我们还观察到与凋亡调控相关的差异表达基因,如 BCL2L1、CASP7、CDKN1A、E2F2、ITPR、CD274、TNFAIP6、TNFRSF25、TNFRSF13C、TNFRSF17。
我们的结果表明,参与免疫反应和凋亡调控信号通路的基因表达改变的组合可能直接导致 BS 观察到的主要特征,如反复感染、生长发育迟缓以及癌症风险高。对其他不稳定性综合征的转录组研究可以更准确地分析与基因组不稳定性相关的相关基因相互作用。这是首次通过 RNA-seq 描述 BS 患者免疫相关差异基因表达谱。