Proteomic analysis reveals chromatin remodeling as a potential therapeutical target in neuroblastoma

Abstract Background Neuroblastoma (NB) is the most common solid tumor in children, characterized by high recurrence rates, drug resistance, and significant mortality. Methods In this study, we analyzed the proteomic profiles of NB tissue samples alongside other pathological categories, including gan...

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Main Authors: Zan Liu, Zitong Zhao, Longlong Xie, Zhenghui Xiao, Ming Li, Yong Li, Ting Luo
Format: Article
Language:English
Published: BMC 2025-02-01
Series:Journal of Translational Medicine
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Online Access:https://doi.org/10.1186/s12967-025-06298-5
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author Zan Liu
Zitong Zhao
Longlong Xie
Zhenghui Xiao
Ming Li
Yong Li
Ting Luo
author_facet Zan Liu
Zitong Zhao
Longlong Xie
Zhenghui Xiao
Ming Li
Yong Li
Ting Luo
author_sort Zan Liu
collection DOAJ
description Abstract Background Neuroblastoma (NB) is the most common solid tumor in children, characterized by high recurrence rates, drug resistance, and significant mortality. Methods In this study, we analyzed the proteomic profiles of NB tissue samples alongside other pathological categories, including ganglioneuroma (GN) and ganglioneuroblastoma (GNB). Using weighted gene co-expression network analysis (WGCNA), the core prognostic gene models associated with histopathology of NB were identified. Furthermore, by mapping our core prognostic gene models onto drug-perturbed transcriptome profiles from the L1000FWD and CMap databases, repurposing drug candidates were screened and validated for NB. Results Our proteomic analysis reveals that pathways associated with the cell cycle and DNA replication are significantly upregulated in NB, while oxidative phosphorylation, pyruvate metabolism, and the TCA cycle are notably downregulated compared to GNB and GN. By applying WGCNA, we identified a core prognostic gene model strongly associated with the unfavorable subtype and high MKI of NB and primarily related to chromatin binding and mRNA metabolic process. Protein–protein interaction network analysis identified 15 hub genes in this core prognostic module: SMARCA4, SMARCA5, SMARCC2, SMARCC1, PBRM1, BRD3, ARID1A, BRD2, ARID1B, KDM1A, TP53BP1, ALYREF, CBX1, SF3B1, and ADNP, which mainly related to chromatin remodeling. Notably, SMARCA4 and ALYREF are also high-risk genes of mortality and validated as potential prognostic biomarkers for NB. Through repurposing drugs screening, mocetinostat and clofarabine were validated as effective treatments in two NB cell lines. Conclusion Mocetinostat and clofarabine offer valuable insights for the development of novel targeted therapies in neuroblastoma.
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spelling doaj-art-60d73f06a5614c1bb6af87aa3eae911d2025-08-20T03:03:45ZengBMCJournal of Translational Medicine1479-58762025-02-0123111110.1186/s12967-025-06298-5Proteomic analysis reveals chromatin remodeling as a potential therapeutical target in neuroblastomaZan Liu0Zitong Zhao1Longlong Xie2Zhenghui Xiao3Ming Li4Yong Li5Ting Luo6Department of Pediatric Surgery, Clinical Research Center for Pediatric Solid Tumors in Hunan Province, Hunan Provincial Key Laboratory of Pediatric Orthopedics, The Affiliated Children’s Hospital of Xiangya School of Medicine, Central South University (Hunan Children’s Hospital)Center of Reproductive Medicine, Hunan Provincial Key Laboratory of Regional Hereditary Birth Defects Prevention and Control, Changsha Hospital for Maternal and Child Health Care of Hunan Normal UniversityDepartment of Radiology, The Affiliated Children’s Hospital of Xiangya School of Medicine, Central South University (Hunan Children’s Hospital)Emergency Center of Hunan Children’s Hospital, The Affiliated Children’s Hospital of Xiangya School of Medicine, Central South UniversityDepartment of Pediatric Surgery, Clinical Research Center for Pediatric Solid Tumors in Hunan Province, Hunan Provincial Key Laboratory of Pediatric Orthopedics, The Affiliated Children’s Hospital of Xiangya School of Medicine, Central South University (Hunan Children’s Hospital)Department of Pediatric Surgery, Clinical Research Center for Pediatric Solid Tumors in Hunan Province, Hunan Provincial Key Laboratory of Pediatric Orthopedics, The Affiliated Children’s Hospital of Xiangya School of Medicine, Central South University (Hunan Children’s Hospital)Pediatrics Research Institute, The Affiliated Children’s Hospital of Xiangya School of Medicine, Central South University (Hunan Children’s Hospital)Abstract Background Neuroblastoma (NB) is the most common solid tumor in children, characterized by high recurrence rates, drug resistance, and significant mortality. Methods In this study, we analyzed the proteomic profiles of NB tissue samples alongside other pathological categories, including ganglioneuroma (GN) and ganglioneuroblastoma (GNB). Using weighted gene co-expression network analysis (WGCNA), the core prognostic gene models associated with histopathology of NB were identified. Furthermore, by mapping our core prognostic gene models onto drug-perturbed transcriptome profiles from the L1000FWD and CMap databases, repurposing drug candidates were screened and validated for NB. Results Our proteomic analysis reveals that pathways associated with the cell cycle and DNA replication are significantly upregulated in NB, while oxidative phosphorylation, pyruvate metabolism, and the TCA cycle are notably downregulated compared to GNB and GN. By applying WGCNA, we identified a core prognostic gene model strongly associated with the unfavorable subtype and high MKI of NB and primarily related to chromatin binding and mRNA metabolic process. Protein–protein interaction network analysis identified 15 hub genes in this core prognostic module: SMARCA4, SMARCA5, SMARCC2, SMARCC1, PBRM1, BRD3, ARID1A, BRD2, ARID1B, KDM1A, TP53BP1, ALYREF, CBX1, SF3B1, and ADNP, which mainly related to chromatin remodeling. Notably, SMARCA4 and ALYREF are also high-risk genes of mortality and validated as potential prognostic biomarkers for NB. Through repurposing drugs screening, mocetinostat and clofarabine were validated as effective treatments in two NB cell lines. Conclusion Mocetinostat and clofarabine offer valuable insights for the development of novel targeted therapies in neuroblastoma.https://doi.org/10.1186/s12967-025-06298-5NeuroblastomaProteomicsChromatin-remodelingSMARCA4
spellingShingle Zan Liu
Zitong Zhao
Longlong Xie
Zhenghui Xiao
Ming Li
Yong Li
Ting Luo
Proteomic analysis reveals chromatin remodeling as a potential therapeutical target in neuroblastoma
Journal of Translational Medicine
Neuroblastoma
Proteomics
Chromatin-remodeling
SMARCA4
title Proteomic analysis reveals chromatin remodeling as a potential therapeutical target in neuroblastoma
title_full Proteomic analysis reveals chromatin remodeling as a potential therapeutical target in neuroblastoma
title_fullStr Proteomic analysis reveals chromatin remodeling as a potential therapeutical target in neuroblastoma
title_full_unstemmed Proteomic analysis reveals chromatin remodeling as a potential therapeutical target in neuroblastoma
title_short Proteomic analysis reveals chromatin remodeling as a potential therapeutical target in neuroblastoma
title_sort proteomic analysis reveals chromatin remodeling as a potential therapeutical target in neuroblastoma
topic Neuroblastoma
Proteomics
Chromatin-remodeling
SMARCA4
url https://doi.org/10.1186/s12967-025-06298-5
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AT longlongxie proteomicanalysisrevealschromatinremodelingasapotentialtherapeuticaltargetinneuroblastoma
AT zhenghuixiao proteomicanalysisrevealschromatinremodelingasapotentialtherapeuticaltargetinneuroblastoma
AT mingli proteomicanalysisrevealschromatinremodelingasapotentialtherapeuticaltargetinneuroblastoma
AT yongli proteomicanalysisrevealschromatinremodelingasapotentialtherapeuticaltargetinneuroblastoma
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