Oncogenic function and transcriptional dynamics of MYCN in liver tumorigenesis

X Xian-Yang Qin (Laboratory for Cellular Function Conversion Technology, RIKEN Center for Integrative Medical Sciences) Y Yali Xu (State Key Laboratory of Drug Research) H Hricha Mishra (Laboratory for Cellular Function Conversion Technology, RIKEN Center for Integrative Medical Sciences) Y Yohei Shirakami (Department of Gastroenterology, Graduate School of Medicine, Gifu University) S Shiou-Hwei Yeh (Department of Microbiology, National Taiwan University College of Medicine) C Chiao-Ling Li (Department of Microbiology, National Taiwan University College of Medicine) K Kazushi Numata (Gastroenterological Center, Yokohama City University Medical Center) Y Yusuke Suenaga F Feifei Wei (Division of Cancer Immunotherapy, Kanagawa Cancer Center Research Institute) R Reiko Ando (Support Unit for Bio-Material Analysis, Research Resources Division, RIKEN Center for Brain Science) H Hajime Nishimura (Laboratory for Cellular Function Conversion Technology, RIKEN Center for Integrative Medical Sciences) E Erina Furuhata (Laboratory for Cellular Function Conversion Technology, RIKEN Center for Integrative Medical Sciences) S Shiori Maeda (Laboratory for Cellular Function Conversion Technology, RIKEN Center for Integrative Medical Sciences) Y Yutaka Furutani (Department of Laboratory Medicine, The Jikei University School of Medicine) K Kaori Yanaka (Department of Laboratory Medicine, The Jikei University School of Medicine) M Masahiro Yamamoto (Department of Immunoparasitology, Research Institute for Microbial Diseases, Osaka University) M Masanori Goto (Division of Tumor Pathology, Department of Pathology, Asahikawa Medical University) A Akira Takasawa (Division of Tumor Pathology, Department of Pathology, Asahikawa Medical University) Y Yuji Nishikawa (Division of Tumor Pathology, Department of Pathology, Asahikawa Medical University) H Hiroyuki Tomita (Department of Tumor Pathology, Gifu University Graduate School of Medicine) L Luc Gailhouste (Laboratory for Brain Development and Disorders, RIKEN Center for Brain Science) T Tomokazu Matsuura (Department of Laboratory Medicine, The Jikei University School of Medicine) P Pei-Jer Chen (Graduate Institute of Clinical Medicine, Department of Internal Medicine, National Taiwan University College of Medicine and Hospital) M Masahito Shimizu (Department of Gastroenterology, Graduate School of Medicine, Gifu University) Y Yoshitaka Hippo (Laboratory of Evolutionary Oncology, Chiba Cancer Center Research Institute) H Harukazu Suzuki (Laboratory for Cellular Function Conversion Technology, RIKEN Center for Integrative Medical Sciences)

Abstract

Hepatocellular carcinoma (HCC) is a leading cause of cancer-related mortality. It is often diagnosed at advanced stages and characterized by high recurrence rates. Although chronic liver inflammation and metabolic dysfunction are established contributors to tumorigenesis, the molecular mechanisms that link microenvironmental stress to malignant transformation remain poorly understood. MYCN, a proto-oncogenic transcription factor, has emerged as a potential biomarker of cancer stemness. However, its role in hepatocarcinogenesis remains unclear. In this study, we elucidated the oncogenic role of MYCN and its dynamic transcriptional regulation during liver tumorigenesis. Using a hydrodynamic tail vein injection-based transposon system in mice, we demonstrated that MYCN overexpression synergizes with AKT activation to promote liver tumorigenesis. Transcriptomic profiling revealed that MYCN-driven tumors exhibited features of human HCC subtypes enriched in stress-adaptive transcriptional programs. Time-resolved spatial transcriptomics further uncovered a MYCN-enriched niche characterized by epithelial–mesenchymal transition (EMT) and Wnt/β-catenin signaling, which expanded during tumor progression and was spatially proximate to transformed malignant cells. To translate these findings to human HCC, we developed a machine learning-based MYCN niche score and validated its clinical relevance across multiple human HCC cohorts. This score reliably predicted recurrence risk and identified EMT-prone microenvironments, with stronger predictive performance in nontumor tissues, suggesting its potential in detecting precancerous niches predisposed to de novo tumorigenesis. Collectively, our findings establish MYCN as a functional driver and spatial marker of tumor-promoting microenvironments in liver tumorigenesis; additionally, we propose a clinically actionable strategy to identify high-risk patients through transcriptomic profiling of nontumor liver tissue.

Article Details

Volume / Issue Vol. 123, Issue 8
Published February 24, 2026
ISSN 0027-8424
Publisher National Academy of Sciences

Authors (26)

X

Xian-Yang Qin

Laboratory for Cellular Function Conversion Technology, RIKEN Center for Integrative Medical Sciences

Y

Yali Xu

State Key Laboratory of Drug Research

H

Hricha Mishra

Laboratory for Cellular Function Conversion Technology, RIKEN Center for Integrative Medical Sciences

Y

Yohei Shirakami

Department of Gastroenterology, Graduate School of Medicine, Gifu University

S

Shiou-Hwei Yeh

Department of Microbiology, National Taiwan University College of Medicine

C

Chiao-Ling Li

Department of Microbiology, National Taiwan University College of Medicine

K

Kazushi Numata

Gastroenterological Center, Yokohama City University Medical Center

Y

Yusuke Suenaga

F

Feifei Wei

Division of Cancer Immunotherapy, Kanagawa Cancer Center Research Institute

R

Reiko Ando

Support Unit for Bio-Material Analysis, Research Resources Division, RIKEN Center for Brain Science

H

Hajime Nishimura

Laboratory for Cellular Function Conversion Technology, RIKEN Center for Integrative Medical Sciences

E

Erina Furuhata

Laboratory for Cellular Function Conversion Technology, RIKEN Center for Integrative Medical Sciences

S

Shiori Maeda

Laboratory for Cellular Function Conversion Technology, RIKEN Center for Integrative Medical Sciences

Y

Yutaka Furutani

Department of Laboratory Medicine, The Jikei University School of Medicine

K

Kaori Yanaka

Department of Laboratory Medicine, The Jikei University School of Medicine

M

Masahiro Yamamoto

Department of Immunoparasitology, Research Institute for Microbial Diseases, Osaka University

M

Masanori Goto

Division of Tumor Pathology, Department of Pathology, Asahikawa Medical University

A

Akira Takasawa

Division of Tumor Pathology, Department of Pathology, Asahikawa Medical University

Y

Yuji Nishikawa

Division of Tumor Pathology, Department of Pathology, Asahikawa Medical University

H

Hiroyuki Tomita

Department of Tumor Pathology, Gifu University Graduate School of Medicine

L

Luc Gailhouste

Laboratory for Brain Development and Disorders, RIKEN Center for Brain Science

T

Tomokazu Matsuura

Department of Laboratory Medicine, The Jikei University School of Medicine

P

Pei-Jer Chen

Graduate Institute of Clinical Medicine, Department of Internal Medicine, National Taiwan University College of Medicine and Hospital

M

Masahito Shimizu

Department of Gastroenterology, Graduate School of Medicine, Gifu University

Y

Yoshitaka Hippo

Laboratory of Evolutionary Oncology, Chiba Cancer Center Research Institute

H

Harukazu Suzuki

Laboratory for Cellular Function Conversion Technology, RIKEN Center for Integrative Medical Sciences