Identification of Crucial Genes Associated With MYCN‐Driven Neuroblastoma Based on Single‐Cell Analysis and Machine Learning

ABSTRACT Background Neuroblastoma (NB) with MYCN amplification is strongly correlated with high‐risk stratification and poor prognosis. However, the underlying mechanisms remain incompletely understood. Elucidating these pathways is critical for advancing personalized treatments for MYCN‐driven NB....

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Main Authors: Jiasi Zhang, Yichen Lei, Yaqin Wang, Wen Yu, Xiaoyan Zhao, Yongbing Zhu, Dedong Zhang, Siying Liu, Aiguo Liu
Format: Article
Language:English
Published: Wiley 2025-07-01
Series:Cancer Medicine
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Online Access:https://doi.org/10.1002/cam4.71008
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author Jiasi Zhang
Yichen Lei
Yaqin Wang
Wen Yu
Xiaoyan Zhao
Yongbing Zhu
Dedong Zhang
Siying Liu
Aiguo Liu
author_facet Jiasi Zhang
Yichen Lei
Yaqin Wang
Wen Yu
Xiaoyan Zhao
Yongbing Zhu
Dedong Zhang
Siying Liu
Aiguo Liu
author_sort Jiasi Zhang
collection DOAJ
description ABSTRACT Background Neuroblastoma (NB) with MYCN amplification is strongly correlated with high‐risk stratification and poor prognosis. However, the underlying mechanisms remain incompletely understood. Elucidating these pathways is critical for advancing personalized treatments for MYCN‐driven NB. Methods We performed single‐cell transcriptomic analysis comparing NB samples with and without MYCN. Key genes were then identified using machine learning based random survival forest (RSF) and nomogram analyses. The influence of key genes on immune infiltration and molecular mechanisms driving NB progression were further investigated. Finally, we visualized the expression levels and global function of these genes in single‐cell datasets and validated their expression in patient samples through RT‐qPCR. Results Single‐cell transcriptome analysis of GSE218450 identified marker genes specific to NB cells. RSF and nomogram analyses revealed that overexpression of CKB, PCSK1N, OTUB1, and VGF is associated with poor prognosis, whereas upregulation of NTRK3 indicates a favorable prognosis. These genes are significantly associated with immune cell infiltration and play an important role in modulating the immune microenvironment. Pathway analysis further showed that these genes influence critical signaling pathways, including the Wnt pathway, and interact with tumor‐related genes. Additionally, we confirmed that CKB and PCSK1N are positively correlated with MYCN in NB cell lines and are significantly overexpressed in MYCN‐amplified NB patients. Conclusions Our results provide molecular insights into the transcriptional changes associated with MYCN amplification in NB. In particular, the identification of CKB and PCSK1N suggests their potential role in driving tumor progression, making them promising targets for novel treatments in MYCN‐driven NB.
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spelling doaj-art-ccdcce0669f94f6aafb8baad72d2b35f2025-08-20T02:54:53ZengWileyCancer Medicine2045-76342025-07-011413n/an/a10.1002/cam4.71008Identification of Crucial Genes Associated With MYCN‐Driven Neuroblastoma Based on Single‐Cell Analysis and Machine LearningJiasi Zhang0Yichen Lei1Yaqin Wang2Wen Yu3Xiaoyan Zhao4Yongbing Zhu5Dedong Zhang6Siying Liu7Aiguo Liu8Department of Pediatric Tongji Hospital, Tongji Medical College, Huazhong University of Science and Technology Wuhan ChinaDepartment of Pediatric Tongji Hospital, Tongji Medical College, Huazhong University of Science and Technology Wuhan ChinaDepartment of Pediatric Tongji Hospital, Tongji Medical College, Huazhong University of Science and Technology Wuhan ChinaDepartment of Pediatric Tongji Hospital, Tongji Medical College, Huazhong University of Science and Technology Wuhan ChinaDepartment of Pediatric Tongji Hospital, Tongji Medical College, Huazhong University of Science and Technology Wuhan ChinaDepartment of Pediatric Tongji Hospital, Tongji Medical College, Huazhong University of Science and Technology Wuhan ChinaDepartment of Pediatric Tongji Hospital, Tongji Medical College, Huazhong University of Science and Technology Wuhan ChinaDepartment of Pediatric Tongji Hospital, Tongji Medical College, Huazhong University of Science and Technology Wuhan ChinaDepartment of Pediatric Tongji Hospital, Tongji Medical College, Huazhong University of Science and Technology Wuhan ChinaABSTRACT Background Neuroblastoma (NB) with MYCN amplification is strongly correlated with high‐risk stratification and poor prognosis. However, the underlying mechanisms remain incompletely understood. Elucidating these pathways is critical for advancing personalized treatments for MYCN‐driven NB. Methods We performed single‐cell transcriptomic analysis comparing NB samples with and without MYCN. Key genes were then identified using machine learning based random survival forest (RSF) and nomogram analyses. The influence of key genes on immune infiltration and molecular mechanisms driving NB progression were further investigated. Finally, we visualized the expression levels and global function of these genes in single‐cell datasets and validated their expression in patient samples through RT‐qPCR. Results Single‐cell transcriptome analysis of GSE218450 identified marker genes specific to NB cells. RSF and nomogram analyses revealed that overexpression of CKB, PCSK1N, OTUB1, and VGF is associated with poor prognosis, whereas upregulation of NTRK3 indicates a favorable prognosis. These genes are significantly associated with immune cell infiltration and play an important role in modulating the immune microenvironment. Pathway analysis further showed that these genes influence critical signaling pathways, including the Wnt pathway, and interact with tumor‐related genes. Additionally, we confirmed that CKB and PCSK1N are positively correlated with MYCN in NB cell lines and are significantly overexpressed in MYCN‐amplified NB patients. Conclusions Our results provide molecular insights into the transcriptional changes associated with MYCN amplification in NB. In particular, the identification of CKB and PCSK1N suggests their potential role in driving tumor progression, making them promising targets for novel treatments in MYCN‐driven NB.https://doi.org/10.1002/cam4.71008MYCN amplificationneuroblastomanomogram modelrandom survival forestssingle‐cell transcriptome
spellingShingle Jiasi Zhang
Yichen Lei
Yaqin Wang
Wen Yu
Xiaoyan Zhao
Yongbing Zhu
Dedong Zhang
Siying Liu
Aiguo Liu
Identification of Crucial Genes Associated With MYCN‐Driven Neuroblastoma Based on Single‐Cell Analysis and Machine Learning
Cancer Medicine
MYCN amplification
neuroblastoma
nomogram model
random survival forests
single‐cell transcriptome
title Identification of Crucial Genes Associated With MYCN‐Driven Neuroblastoma Based on Single‐Cell Analysis and Machine Learning
title_full Identification of Crucial Genes Associated With MYCN‐Driven Neuroblastoma Based on Single‐Cell Analysis and Machine Learning
title_fullStr Identification of Crucial Genes Associated With MYCN‐Driven Neuroblastoma Based on Single‐Cell Analysis and Machine Learning
title_full_unstemmed Identification of Crucial Genes Associated With MYCN‐Driven Neuroblastoma Based on Single‐Cell Analysis and Machine Learning
title_short Identification of Crucial Genes Associated With MYCN‐Driven Neuroblastoma Based on Single‐Cell Analysis and Machine Learning
title_sort identification of crucial genes associated with mycn driven neuroblastoma based on single cell analysis and machine learning
topic MYCN amplification
neuroblastoma
nomogram model
random survival forests
single‐cell transcriptome
url https://doi.org/10.1002/cam4.71008
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