Ito Fumiaki, Li Ziyuan, Minakhin Leonid, Khant Htet A, Pomerantz Richard T, Chen Xiaojiang S
Molecular and Computational Biology, Department of Biological Sciences and Chemistry, University of Southern California, Los Angeles, California, 90089, USA.
Department of Microbiology, Immunology and Molecular Genetics.
bioRxiv. 2024 Jun 7:2024.06.07.597860. doi: 10.1101/2024.06.07.597860.
DNA double-strand breaks (DSBs) present a critical threat to genomic integrity, often precipitating genomic instability and oncogenesis. Repair of DSBs predominantly occurs through homologous recombination (HR) and non-homologous end joining (NHEJ). In HR-deficient cells, DNA polymerase theta (Polθ) becomes critical for DSB repair via microhomology-mediated end joining (MMEJ), also termed theta-mediated end joining (TMEJ). Thus, Polθ is synthetically lethal with BRCA1/2 and other HR factors, underscoring its potential as a therapeutic target in HR-deficient cancers. However, the molecular mechanisms governing Polθ-mediated MMEJ remain poorly understood. Here we present a series of cryo-electron microscopy structures of the Polθ helicase domain (Polθ-hel) in complex with DNA containing 3'-overhang. The structures reveal the sequential conformations adopted by Polθ-hel during the critical phases of DNA binding, microhomology searching, and microhomology annealing. The stepwise conformational changes within the Polθ-hel subdomains and its functional dimeric state are pivotal for aligning the 3'-overhangs, facilitating the microhomology search and subsequent annealing necessary for DSB repair via MMEJ. Our findings illustrate the essential molecular switches within Polθ-hel that orchestrate the MMEJ process in DSB repair, laying the groundwork for the development of targeted therapies against the Polθ-hel.
DNA双链断裂(DSBs)对基因组完整性构成了严重威胁,常常引发基因组不稳定和肿瘤发生。DSBs的修复主要通过同源重组(HR)和非同源末端连接(NHEJ)进行。在HR缺陷细胞中,DNA聚合酶θ(Polθ)通过微同源性介导的末端连接(MMEJ,也称为θ介导的末端连接,TMEJ)对DSB修复变得至关重要。因此,Polθ与BRCA1/2及其他HR因子具有合成致死性,这凸显了其作为HR缺陷型癌症治疗靶点的潜力。然而,目前对于Polθ介导的MMEJ的分子机制仍知之甚少。在此,我们展示了一系列Polθ解旋酶结构域(Polθ-hel)与含3'突出端的DNA复合物的冷冻电子显微镜结构。这些结构揭示了Polθ-hel在DNA结合、微同源性搜索和微同源性退火的关键阶段所采取的连续构象。Polθ-hel亚结构域内逐步的构象变化及其功能性二聚体状态对于对齐3'突出端、促进微同源性搜索以及随后通过MMEJ进行DSB修复所需的退火过程至关重要。我们的研究结果阐明了Polθ-hel内协调DSB修复中MMEJ过程的关键分子开关,为开发针对Polθ-hel的靶向疗法奠定了基础。