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[骨髓增生异常综合征及相关疾病中的基因组畸变]

[Genomic aberrations in myelodysplastic syndromes and related disorders].

作者信息

Makishima Hideki

机构信息

Pathology and Tumor Biology, Graduate School of Medicine, Kyoto University.

出版信息

Rinsho Ketsueki. 2019;60(6):600-609. doi: 10.11406/rinketsu.60.600.

Abstract

Myelodysplastic syndromes (MDS) and myelodysplastic/myeloproliferative neoplasms (MDS/MPN) are heterogeneous myeloid neoplasms that frequently evolve into secondary acute myeloid leukemia (sAML). Recent progress in next-generation sequencing technologies has allowed us to discover frequent mutations throughout the coding regions of MDS, MDS/MPN, and sAML, subsequently providing information on more than 60 driver genes in these diseases. As shown by many study groups recently, such driver mutations are acquired in a gene-specific fashion. DDX41 and SAMD9/SAMD9L mutations are observed in germline cells long before MDS presentation. In blood samples from healthy elderly individuals, somatic DNMT3A, TET2, and ASXL1 mutations are detected as age-related clonal hematopoiesis and supposed to be a risk factor for hematological neoplasms. Recent reports on MDS have shown that mutations in genes such as NRAS and FLT3, designated as Type I genes, were significantly associated with leukemic evolution. Another type (Type II) of genes, including RUNX1 and GATA2, has been shown to be related to the progression from low-risk to high-risk MDS. These driver mutations are significantly concomitant during disease progression. Overall, various types of driver mutations are sequentially acquired in MDS, accounting for the heterogeneity of these disorders.

摘要

骨髓增生异常综合征(MDS)和骨髓增生异常/骨髓增殖性肿瘤(MDS/MPN)是异质性髓系肿瘤,常进展为继发性急性髓系白血病(sAML)。新一代测序技术的最新进展使我们能够在MDS、MDS/MPN和sAML的编码区域发现频繁的突变,随后提供了有关这些疾病中60多个驱动基因的信息。正如许多研究小组最近所表明的,此类驱动突变是以基因特异性方式获得的。在MDS出现之前很久就在生殖细胞中观察到DDX41和SAMD9/SAMD9L突变。在健康老年人的血液样本中,体细胞DNMT3A、TET2和ASXL1突变被检测为与年龄相关的克隆性造血,被认为是血液系统肿瘤的危险因素。最近关于MDS的报告表明,NRAS和FLT3等基因(称为I型基因)的突变与白血病进展显著相关。另一类(II型)基因,包括RUNX1和GATA2,已被证明与MDS从低危向高危进展有关。这些驱动突变在疾病进展过程中显著伴随出现。总体而言,MDS中依次获得各种类型的驱动突变,这解释了这些疾病的异质性。

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