Integrative molecular subtyping and diagnostic model construction for Moyamoya disease based on diverse programmed cell death gene patterns

Abstract Background The role of cell death in the pathogenesis of Moyamoya disease (MMD) remains unclear. Methods Gene expression data from two publicly available MMD datasets (GSE157628 and GSE189993) were integrated. Differential expression analysis of cell death-related genes was performed, follo...

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Main Authors: Qikai Tang, Chenfeng Ma, Hu Li, Jiaheng Xie, Weiqi Bian, Qingyu Lu, Zeyu Wan, Wei Wu
Format: Article
Language:English
Published: BMC 2025-06-01
Series:Orphanet Journal of Rare Diseases
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Online Access:https://doi.org/10.1186/s13023-025-03816-y
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author Qikai Tang
Chenfeng Ma
Hu Li
Jiaheng Xie
Weiqi Bian
Qingyu Lu
Zeyu Wan
Wei Wu
author_facet Qikai Tang
Chenfeng Ma
Hu Li
Jiaheng Xie
Weiqi Bian
Qingyu Lu
Zeyu Wan
Wei Wu
author_sort Qikai Tang
collection DOAJ
description Abstract Background The role of cell death in the pathogenesis of Moyamoya disease (MMD) remains unclear. Methods Gene expression data from two publicly available MMD datasets (GSE157628 and GSE189993) were integrated. Differential expression analysis of cell death-related genes was performed, followed by immune cell infiltration analysis using the CIBERSORT algorithm. Unsupervised clustering identified molecular subgroups within MMD patients, and these were further analyzed using Gene Set Variation Analysis (GSVA) and Weighted Gene Co-expression Network Analysis (WGCNA). Diagnostic models were constructed using machine learning algorithms, and key model genes were validated by qRT-PCR in arterial samples from MMD patients. Results Significant differences in gene expression were observed, with immune cell infiltration analysis showing differences in T follicular helper cells, activated dendritic cells, resting and activated mast cells, and eosinophils. Unsupervised clustering identified two distinct patient groups, and differential gene expression revealed upregulation of genes like ADRB2, FGR, ICAM1, IL1B, DDIT4, CXCL1, CASP4, G0S2, CYP1B1, and CD74 in C2 cluster. Weighted gene co-expression network analysis (WGCNA) identified key genes in the most correlated module. Machine learning models (RF, SVM, GLM, XGB) were constructed, with the SVM model showing the best performance (AUC = 1.000). A nomogram based on the SVM model demonstrated high predictive accuracy. Enrichment analysis revealed that key genes were involved in apoptosis and antigen processing, with strong diagnostic performance confirmed by ROC curves. PCR analysis revealed that model genes RGS1, MUC1, KCNA2, TAC1, and SOST were all up-regulated in MMD group. Conclusions This study provides novel insights into the molecular landscape of MMD, highlighting the importance of cell death-related pathways and immune responses. The identified biomarkers and molecular subgroups offer potential targets for therapeutic intervention and improved diagnostic strategies in MMD.
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spelling doaj-art-0ba2e8019dc443c18c6d4da2ba9019372025-08-20T02:05:42ZengBMCOrphanet Journal of Rare Diseases1750-11722025-06-0120111410.1186/s13023-025-03816-yIntegrative molecular subtyping and diagnostic model construction for Moyamoya disease based on diverse programmed cell death gene patternsQikai Tang0Chenfeng Ma1Hu Li2Jiaheng Xie3Weiqi Bian4Qingyu Lu5Zeyu Wan6Wei Wu7Department of Neurosurgery, Jiangsu Province Hospital, The First Affiliated Hospital of Nanjing Medical UniversityDepartment of Neurosurgery, Jiangsu Province Hospital, The First Affiliated Hospital of Nanjing Medical UniversityDepartment of Neurosurgery, People’s Hospital of Ganyu DistrictDepartment of Plastic Surgery, Xiangya Hospital, Central South UniversityDepartment of Neurosurgery, Jiangsu Province Hospital, The First Affiliated Hospital of Nanjing Medical UniversityDepartment of Neurosurgery, Jiangsu Province Hospital, The First Affiliated Hospital of Nanjing Medical UniversityDepartment of Neurosurgery, Jiangsu Province Hospital, The First Affiliated Hospital of Nanjing Medical UniversityDepartment of Neurosurgery, Jiangsu Province Hospital, The First Affiliated Hospital of Nanjing Medical UniversityAbstract Background The role of cell death in the pathogenesis of Moyamoya disease (MMD) remains unclear. Methods Gene expression data from two publicly available MMD datasets (GSE157628 and GSE189993) were integrated. Differential expression analysis of cell death-related genes was performed, followed by immune cell infiltration analysis using the CIBERSORT algorithm. Unsupervised clustering identified molecular subgroups within MMD patients, and these were further analyzed using Gene Set Variation Analysis (GSVA) and Weighted Gene Co-expression Network Analysis (WGCNA). Diagnostic models were constructed using machine learning algorithms, and key model genes were validated by qRT-PCR in arterial samples from MMD patients. Results Significant differences in gene expression were observed, with immune cell infiltration analysis showing differences in T follicular helper cells, activated dendritic cells, resting and activated mast cells, and eosinophils. Unsupervised clustering identified two distinct patient groups, and differential gene expression revealed upregulation of genes like ADRB2, FGR, ICAM1, IL1B, DDIT4, CXCL1, CASP4, G0S2, CYP1B1, and CD74 in C2 cluster. Weighted gene co-expression network analysis (WGCNA) identified key genes in the most correlated module. Machine learning models (RF, SVM, GLM, XGB) were constructed, with the SVM model showing the best performance (AUC = 1.000). A nomogram based on the SVM model demonstrated high predictive accuracy. Enrichment analysis revealed that key genes were involved in apoptosis and antigen processing, with strong diagnostic performance confirmed by ROC curves. PCR analysis revealed that model genes RGS1, MUC1, KCNA2, TAC1, and SOST were all up-regulated in MMD group. Conclusions This study provides novel insights into the molecular landscape of MMD, highlighting the importance of cell death-related pathways and immune responses. The identified biomarkers and molecular subgroups offer potential targets for therapeutic intervention and improved diagnostic strategies in MMD.https://doi.org/10.1186/s13023-025-03816-yMoyamoya diseaseBioinformaticsWGCNARNA-sequencingBiomarker
spellingShingle Qikai Tang
Chenfeng Ma
Hu Li
Jiaheng Xie
Weiqi Bian
Qingyu Lu
Zeyu Wan
Wei Wu
Integrative molecular subtyping and diagnostic model construction for Moyamoya disease based on diverse programmed cell death gene patterns
Orphanet Journal of Rare Diseases
Moyamoya disease
Bioinformatics
WGCNA
RNA-sequencing
Biomarker
title Integrative molecular subtyping and diagnostic model construction for Moyamoya disease based on diverse programmed cell death gene patterns
title_full Integrative molecular subtyping and diagnostic model construction for Moyamoya disease based on diverse programmed cell death gene patterns
title_fullStr Integrative molecular subtyping and diagnostic model construction for Moyamoya disease based on diverse programmed cell death gene patterns
title_full_unstemmed Integrative molecular subtyping and diagnostic model construction for Moyamoya disease based on diverse programmed cell death gene patterns
title_short Integrative molecular subtyping and diagnostic model construction for Moyamoya disease based on diverse programmed cell death gene patterns
title_sort integrative molecular subtyping and diagnostic model construction for moyamoya disease based on diverse programmed cell death gene patterns
topic Moyamoya disease
Bioinformatics
WGCNA
RNA-sequencing
Biomarker
url https://doi.org/10.1186/s13023-025-03816-y
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