Cooke Emmalee W, Zeng Cheng, Nur Suza Mohammad, Jia Yunbo, Huang Aileen, Chen Jiwei, Gao Peidong, Chen Fei Xavier, Jin Fulai, Cao Kaixiang
Department of Biochemistry, Case Western Reserve University.
Case Comprehensive Cancer Center, Case Western Reserve University, 10900 Euclid Ave, Cleveland, OH 44106, USA.
bioRxiv. 2025 May 13:2025.05.08.652740. doi: 10.1101/2025.05.08.652740.
In metazoans, nucleosomes harboring H3K79 methylation (H3K79me) deposited by the histone methyltransferase DOT1L decorate actively transcribed genes. Although DOT1L is implicated in transcription regulation and pathogenesis of human diseases such as leukemia and neurological disorders, the role of H3K79me in these biological processes remains elusive. Here, we reveal a novel functional synergism between H3K79me and H3K36 tri-methylation (H3K36me3), another histone modification enriched at active genes, in regulating gene expression and neural cell fate transition. Simultaneous catalytic inactivation of DOT1L and the H3K36 methyltransferase SETD2 via gene editing leads to the global loss of H3K79me and H3K36me3, hyperactive transcription, and failures in neural differentiation. Interestingly, the loss of H3K79me and H3K36me3 causes increased transcription elongation, gained chromatin accessibility at a group of enhancers, and increased binding of TEAD4 transcription factor and its co-activator YAP1 at these enhancers. Furthermore, YAP-TEAD inhibition partially restores the expression levels of hyperactivated genes upon H3K79me/H3K36me3 loss. Taken together, our study demonstrates a synergistic role of H3K79me and H3K36me3 in regulating transcription and cell fate transition, unveils novel mechanisms underlying such synergism, and provides insight into designing therapies that target diseases driven by misregulation or mutations of DOT1L and/or SETD2.
在多细胞动物中,由组蛋白甲基转移酶DOT1L沉积的携带H3K79甲基化(H3K79me)的核小体修饰活跃转录的基因。尽管DOT1L与转录调控以及白血病和神经疾病等人疾病的发病机制有关,但H3K79me在这些生物学过程中的作用仍不清楚。在这里,我们揭示了H3K79me与H3K36三甲基化(H3K36me3,另一种在活跃基因上富集的组蛋白修饰)在调节基因表达和神经细胞命运转变方面存在新的功能协同作用。通过基因编辑同时催化失活DOT1L和H3K36甲基转移酶SETD2会导致H3K79me和H3K36me3的整体缺失、转录过度活跃以及神经分化失败。有趣的是,H3K79me和H3K36me3的缺失会导致转录延伸增加、一组增强子处的染色质可及性增加,以及TEAD4转录因子及其共激活因子YAP1在这些增强子处的结合增加。此外,YAP - TEAD抑制可部分恢复H3K79me/H3K36me3缺失后过度激活基因的表达水平。综上所述,我们的研究证明了H3K79me和H3K36me3在调节转录和细胞命运转变中的协同作用,揭示了这种协同作用的新机制,并为设计针对由DOT1L和/或SETD2调控异常或突变驱动的疾病的疗法提供了见解。