癌症研究
同源重组
细胞凋亡
生物
前药
化学
癌症
同源染色体
DNA损伤
分子生物学
DNA
泛素
恶性肿瘤
DNA修复
合成致死
细胞生物学
血浆蛋白结合
靶向治疗
作者
Hanyi He,Yangchan Hu,Ye Lu,Mingcong Deng,Qimei Bao,Dan Zu,Yuke Zhong,Chunkai Zhang,Chenlong You,XP Du,Siqi Liu,Yanhua He,M Liu,Liangliang Gao,Xiangzheng Gao,Haidong Liu,Y.‐Z. HUANG,Yuehua Chen,Dade Rong,John A. Tainer
出处
期刊:Cancer Research
[American Association for Cancer Research]
日期:2026-06-18
标识
DOI:10.1158/0008-5472.can-25-4984
摘要
Abstract Gastric cancer (GC) is a lethal malignancy with limited targeted therapeutic options, particularly for HER2-negative disease. Through integrated bioinformatic analysis of multiple GC cohorts, we identified poly(ADP-ribose) glycohydrolase (PARG) as a DNA damage response protein that is frequently overexpressed and associated with poor prognosis. While PARG inhibition as a monotherapy showed limited efficacy, high-throughput screening of FDA-approved drugs revealed a synthetic lethal interaction between PARG inhibition and fluoropyrimidines, including 5-fluorouracil (5-FU). Mechanistically, PARG directly bound the homologous recombination (HR) cofactor RAD51AP1 via its N-terminal domain and removed poly(ADP-ribose) (PAR) chains. The dePARylation prevented RNF169-mediated K48-linked ubiquitination of RAD51AP1, thereby protecting RAD51AP1 from proteasomal degradation. Consequently, loss of PARG destabilized RAD51AP1 and impaired HR repair, resulting in increased sensitivity to fluoropyrimidine-induced DNA lesions. In patient-derived xenograft models, PARG inhibition significantly enhanced the efficacy of both 5-FU and the oral prodrug capecitabine. Moreover, restoring RAD51AP1 expression substantially reversed the chemosensitizing effect conferred by PARG deficiency. Clinically, PARG and RAD51AP1 protein levels were strongly correlated in GC tissues, and their co-expression defined a subset of patients with the worst survival. Collectively, these findings identify a PARylation-ubiquitination switch regulated by PARG that stabilizes RAD51AP1 to enhance HR repair, unveiling the PARG-RAD51AP1 axis as a key determinant of fluoropyrimidine sensitivity and a biomarker-directed target for combination therapy in GC.
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