Stage-Specific Serum Proteomic Signatures Reveal Early Biomarkers and Molecular Pathways in Huntington’s Disease Progression

Background: Huntington’s Disease (HD) is a monogenic neurodegenerative disease resulting in a CAG repeat expansion in the <i>HTT</i> gene. Despite this genetic simplicity, its molecular mechanisms remain highly complex. Methods: In this study, untargeted serum proteomics, bioinformatics...

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Main Authors: Christiana C. Christodoulou, Christiana A. Demetriou, Eleni Zamba-Papanicolaou
Format: Article
Language:English
Published: MDPI AG 2025-08-01
Series:Cells
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Online Access:https://www.mdpi.com/2073-4409/14/15/1195
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author Christiana C. Christodoulou
Christiana A. Demetriou
Eleni Zamba-Papanicolaou
author_facet Christiana C. Christodoulou
Christiana A. Demetriou
Eleni Zamba-Papanicolaou
author_sort Christiana C. Christodoulou
collection DOAJ
description Background: Huntington’s Disease (HD) is a monogenic neurodegenerative disease resulting in a CAG repeat expansion in the <i>HTT</i> gene. Despite this genetic simplicity, its molecular mechanisms remain highly complex. Methods: In this study, untargeted serum proteomics, bioinformatics analysis, biomarker filtering and ELISA validation were implemented to characterize the proteomic landscape across the three HD stages—asymptomatic, early symptomatic and symptomatic advanced—alongside gender/age-matched controls. Results: We identified 84 over-expressed and 118 under-expressed differentially expressed proteins. Enrichment analysis revealed dysregulation in pathways including the complement cascade, LXR/RXR activation and RHOGDI signaling. Biomarker analysis highlighted key proteins with diagnostic potential, including CAP1 (<i>AUC</i> = 0.809), CAPZB (<i>AUC</i> = 0.861), TAGLN2 (<i>AUC</i> = 0.886), THBS1 (<i>AUC</i> = 0.883) and CFH (<i>AUC</i> = 0.948). CAP1 and CAPZB demonstrated robust diagnostic potential in linear mixed-effects models. CAP1 decreased in the asymptomatic stage, suggesting early cytoskeletal disruption, while CAPZB was consistently increased across HD stages. Conclusions: Our findings illuminate the dynamic proteomic and molecular landscape of HD. Future studies should validate these candidates in larger, more diverse cohorts and explore their mechanistic roles in HD pathology and progression.
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spelling doaj-art-c59e33f91a0b4c0a905b68c21c19bb612025-08-20T03:02:48ZengMDPI AGCells2073-44092025-08-011415119510.3390/cells14151195Stage-Specific Serum Proteomic Signatures Reveal Early Biomarkers and Molecular Pathways in Huntington’s Disease ProgressionChristiana C. Christodoulou0Christiana A. Demetriou1Eleni Zamba-Papanicolaou2Neuroepidemiology Department, The Cyprus Institute of Neurology and Genetics, Nicosia 2371, CyprusDepartment of Primary Care and Population Health, University of Nicosia Medical School, Nicosia 2371, CyprusNeuroepidemiology Department, The Cyprus Institute of Neurology and Genetics, Nicosia 2371, CyprusBackground: Huntington’s Disease (HD) is a monogenic neurodegenerative disease resulting in a CAG repeat expansion in the <i>HTT</i> gene. Despite this genetic simplicity, its molecular mechanisms remain highly complex. Methods: In this study, untargeted serum proteomics, bioinformatics analysis, biomarker filtering and ELISA validation were implemented to characterize the proteomic landscape across the three HD stages—asymptomatic, early symptomatic and symptomatic advanced—alongside gender/age-matched controls. Results: We identified 84 over-expressed and 118 under-expressed differentially expressed proteins. Enrichment analysis revealed dysregulation in pathways including the complement cascade, LXR/RXR activation and RHOGDI signaling. Biomarker analysis highlighted key proteins with diagnostic potential, including CAP1 (<i>AUC</i> = 0.809), CAPZB (<i>AUC</i> = 0.861), TAGLN2 (<i>AUC</i> = 0.886), THBS1 (<i>AUC</i> = 0.883) and CFH (<i>AUC</i> = 0.948). CAP1 and CAPZB demonstrated robust diagnostic potential in linear mixed-effects models. CAP1 decreased in the asymptomatic stage, suggesting early cytoskeletal disruption, while CAPZB was consistently increased across HD stages. Conclusions: Our findings illuminate the dynamic proteomic and molecular landscape of HD. Future studies should validate these candidates in larger, more diverse cohorts and explore their mechanistic roles in HD pathology and progression.https://www.mdpi.com/2073-4409/14/15/1195Huntington’s Diseaseproteomicsserum analysisproteinsbioinformaticspathway analysis
spellingShingle Christiana C. Christodoulou
Christiana A. Demetriou
Eleni Zamba-Papanicolaou
Stage-Specific Serum Proteomic Signatures Reveal Early Biomarkers and Molecular Pathways in Huntington’s Disease Progression
Cells
Huntington’s Disease
proteomics
serum analysis
proteins
bioinformatics
pathway analysis
title Stage-Specific Serum Proteomic Signatures Reveal Early Biomarkers and Molecular Pathways in Huntington’s Disease Progression
title_full Stage-Specific Serum Proteomic Signatures Reveal Early Biomarkers and Molecular Pathways in Huntington’s Disease Progression
title_fullStr Stage-Specific Serum Proteomic Signatures Reveal Early Biomarkers and Molecular Pathways in Huntington’s Disease Progression
title_full_unstemmed Stage-Specific Serum Proteomic Signatures Reveal Early Biomarkers and Molecular Pathways in Huntington’s Disease Progression
title_short Stage-Specific Serum Proteomic Signatures Reveal Early Biomarkers and Molecular Pathways in Huntington’s Disease Progression
title_sort stage specific serum proteomic signatures reveal early biomarkers and molecular pathways in huntington s disease progression
topic Huntington’s Disease
proteomics
serum analysis
proteins
bioinformatics
pathway analysis
url https://www.mdpi.com/2073-4409/14/15/1195
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AT christianaademetriou stagespecificserumproteomicsignaturesrevealearlybiomarkersandmolecularpathwaysinhuntingtonsdiseaseprogression
AT elenizambapapanicolaou stagespecificserumproteomicsignaturesrevealearlybiomarkersandmolecularpathwaysinhuntingtonsdiseaseprogression