Department of Hematology, Erasmus MC Cancer Institute, Rotterdam, the Netherlands.
Oncode Institute, Utrecht, the Netherlands.
Blood Cancer Discov. 2024 Sep 3;5(5):303-317. doi: 10.1158/2643-3230.BCD-23-0211.
Although the study of leukemogenesis has traditionally focused on protein-coding genes, the role of enhancer dysregulation is becoming increasingly recognized. The advent of high-throughput sequencing, together with a better understanding of enhancer biology, has revealed how various genetic and epigenetic lesions produce oncogenic enhancers that drive transformation. These aberrations include translocations that lead to enhancer hijacking, point mutations that modulate enhancer activity, and copy number alterations that modify enhancer dosage. In this review, we describe these mechanisms in the context of leukemia and discuss potential therapeutic avenues to target these regulatory elements. Significance: Large-scale sequencing projects have uncovered recurrent gene mutations in leukemia, but the picture remains incomplete: some patients harbor no such aberrations, whereas others carry only a few that are insufficient to bring about transformation on their own. One of the missing pieces is enhancer dysfunction, which only recently has emerged as a critical driver of leukemogenesis. Knowledge of the various mechanisms of enhancer dysregulation is thus key for a complete understanding of leukemia and its causes, as well as the development of targeted therapies in the era of precision medicine.
虽然白血病发生的研究传统上集中在蛋白编码基因上,但增强子失调的作用正越来越受到重视。高通量测序的出现,以及对增强子生物学的更好理解,揭示了各种遗传和表观遗传损伤如何产生致癌增强子,从而驱动转化。这些异常包括导致增强子劫持的易位、调节增强子活性的点突变以及改变增强子剂量的拷贝数改变。在这篇综述中,我们将在白血病的背景下描述这些机制,并讨论针对这些调节元件的潜在治疗途径。意义:大规模测序项目已经揭示了白血病中反复出现的基因突变,但情况仍然不完整:一些患者没有这种异常,而另一些患者只携带少数几个不足以自行引起转化的异常。缺失的部分之一是增强子功能障碍,它最近才成为白血病发生的关键驱动因素。因此,了解增强子失调的各种机制对于全面了解白血病及其病因以及在精准医学时代开发靶向治疗至关重要。