biostudies-literatureChen YLJiangsu province&apos;s science and technology support program (Social Development) projectJiangsu Province Key R&D Program (Social Development) ProjectsJiangsu province's science and technology support program (Social Development)Jiangsu province's science and technology support program (Social Development) projectthe Universities Natural Science Foundation of Jiangsu ProvinceNational Natural Science Foundation of ChinaJiangsu province&apos;s science and technology support programJiangsu Province Key R&amp;D Program (Social Development) ProjectsNatural Science Foundation of Jiangsu Province33https://www.ebi.ac.uk/biostudies/studies/S-EPMC8932231biostudies-literatureUnknown12(1)<h4>Background</h4>Neuroblastoma (NB) is a common extracranial malignancy with high mortality in children. Recently, super-enhancers (SEs) have been reported to play a critical role in the tumorigenesis and development of NB via regulating a wide range of oncogenes Thus, the synthesis and identification of chemical inhibitors specifically targeting SEs are of great urgency for the clinical therapy of NB. This study aimed to characterize the activity of the SEs inhibitor GNE987, which targets BRD4, in NB.<h4>Results</h4>In this study, we found that nanomolar concentrations of GNE987 markedly diminished NB cell proliferation and survival via degrading BRD4. Meanwhile, GNE987 significantly induced NB cell apoptosis and cell cycle arrest. Consistent with in vitro results, GNE987 administrationCell & bioscienceBRD4 inhibitor GNE987 exerts anti-cancer effects by targeting super-enhancers in neuroblastoma.PMC8932231819701638190253482072767819718675200318381802499No.16KJB310014BK20191175BK20190185BE2021657BE2020659BK20190186SBK2019021442BE202165481872845Zhu ZHFang FLi XLTao YFWu DLi ZHXu YYFei XDJiao WYCao HBYang PCZhuo RLi GXie YWang HRJia SQWang JWZhang ZMPan JYang YQian GHFeng CXLiao XMChen YLYu JJfalseBRD4 inhibitor GNE987 exerts anti-cancer effects by targeting super-enhancers in neuroblastoma.<h4>Background</h4>Neuroblastoma (NB) is a common extracranial malignancy with high mortality in children. Recently, super-enhancers (SEs) have been reported to play a critical role in the tumorigenesis and development of NB via regulating a wide range of oncogenes Thus, the synthesis and identification of chemical inhibitors specifically targeting SEs are of great urgency for the clinical therapy of NB. This study aimed to characterize the activity of the SEs inhibitor GNE987, which targets BRD4, in NB.<h4>Results</h4>In this study, we found that nanomolar concentrations of GNE987 markedly diminished NB cell proliferation and survival via degrading BRD4. Meanwhile, GNE987 significantly induced NB cell apoptosis and cell cycle arrest. Consistent with in vitro results, GNE987 administration2022-01-01T00:00:00Z2022 Mar2026-04-08T17:52:21.844Z2025-02-19T05:05:05.883ZS-EPMC89322313530394010.1186/s13578-022-00769-8