Guo Chen-Jun, Bao Xiaojie, Liu Ji-Long
School of Life Science and Technology, ShanghaiTech University, Shanghai, China.
Department of Physiology, Anatomy and Genetics, University of Oxford, Oxford, UK.
Cell Biosci. 2025 Jul 30;15(1):112. doi: 10.1186/s13578-025-01450-6.
CTP synthase (CTPS) is a key enzyme in de novo CTP synthesis, playing a critical role in nucleotide metabolism and cellular proliferation. Human CTPS1 (hCTPS1), one of the two CTPS isoforms, is essential for immune responses and is highly expressed in proliferating cells, making it a promising therapeutic target for immune-related diseases and cancer. Despite its importance, the regulatory mechanisms governing hCTPS1 activity remain poorly understood. Here, we reveal that CTP, the product of CTPS, acts as a key regulator for hCTPS1 filamentation. Using cryo-electron microscopy (cryo-EM), we resolve the high-resolution structure of CTP-bound hCTPS1 filaments, uncovering the molecular details of CTP binding and its role in filament assembly. Importantly, we demonstrate that CTP generated from the enzymatic reaction does not trigger filament disassembly, suggesting a conserved regulatory pattern. Furthermore, by analyzing the binding modes of two distinct CTP-binding pockets, we provide evidence that this filamentation mechanism is evolutionarily conserved across species, particularly in eukaryotic CTPS. Our findings not only elucidate a novel regulatory mechanism of hCTPS1 activity but also deepen the understanding of how metabolic enzymes utilize filamentation as a conserved strategy for functional regulation. This study opens new avenues for targeting hCTPS1 in therapeutic interventions.
胞嘧啶核苷三磷酸合成酶(CTPS)是从头合成CTP的关键酶,在核苷酸代谢和细胞增殖中起关键作用。人CTPS1(hCTPS1)是CTPS的两种同工型之一,对免疫反应至关重要,在增殖细胞中高度表达,使其成为免疫相关疾病和癌症的一个有前景的治疗靶点。尽管其很重要,但hCTPS1活性的调控机制仍知之甚少。在此,我们揭示CTPS的产物CTP作为hCTPS1丝状化的关键调节因子。利用冷冻电子显微镜(cryo-EM),我们解析了结合CTP的hCTPS1丝的高分辨率结构,揭示了CTP结合的分子细节及其在丝组装中的作用。重要的是,我们证明酶促反应产生的CTP不会触发丝的解体,这表明存在一种保守的调控模式。此外,通过分析两个不同CTP结合口袋的结合模式,我们提供证据表明这种丝状化机制在物种间进化保守,尤其是在真核生物的CTPS中。我们的发现不仅阐明了hCTPS1活性的一种新调控机制,还加深了对代谢酶如何利用丝状化作为一种保守的功能调控策略 的理解。这项研究为在治疗干预中靶向hCTPS1开辟了新途径。