Exon-variant interplay and multi-modal evidence identify endocrine dysregulation in severe psychiatric disorders impacting excitatory neurons

Abstract Bipolar disorder (BD), major depressive disorder (MDD), and schizophrenia share genetic architecture, yet their molecular mechanisms remain elusive. Both common and rare genetic variants contribute to neural dysfunction, impacting cognition and behavior. This study investigates the molecula...

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Main Authors: Karolina Worf, Natalie Matosin, Nathalie Gerstner, Anna S. Fröhlich, Anna C. Koller, Franziska Degenhardt, Holger Thiele, Marcella Rietschel, Madhara Udawela, Elizabeth Scarr, Brian Dean, Fabian J. Theis, Nikola S. Mueller, Janine Knauer-Arloth
Format: Article
Language:English
Published: Nature Publishing Group 2025-04-01
Series:Translational Psychiatry
Online Access:https://doi.org/10.1038/s41398-025-03366-8
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author Karolina Worf
Natalie Matosin
Nathalie Gerstner
Anna S. Fröhlich
Anna C. Koller
Franziska Degenhardt
Holger Thiele
Marcella Rietschel
Madhara Udawela
Elizabeth Scarr
Brian Dean
Fabian J. Theis
Nikola S. Mueller
Janine Knauer-Arloth
author_facet Karolina Worf
Natalie Matosin
Nathalie Gerstner
Anna S. Fröhlich
Anna C. Koller
Franziska Degenhardt
Holger Thiele
Marcella Rietschel
Madhara Udawela
Elizabeth Scarr
Brian Dean
Fabian J. Theis
Nikola S. Mueller
Janine Knauer-Arloth
author_sort Karolina Worf
collection DOAJ
description Abstract Bipolar disorder (BD), major depressive disorder (MDD), and schizophrenia share genetic architecture, yet their molecular mechanisms remain elusive. Both common and rare genetic variants contribute to neural dysfunction, impacting cognition and behavior. This study investigates the molecular effects of genetic variants on human cortical single-cell types using a single-exon analysis approach. Integrating exon-level eQTLs (common variants influencing exon expression) and joint exon eQT-Scores (combining polygenic risk scores with exon-level gene expression) from a postmortem psychiatric cohort (BD = 15, MDD = 24, schizophrenia = 68, controls = 62) with schizophrenia-focused rare variant data from the SCHEMA consortium, we identified 110 core genes enriched in pathways including circadian entrainment (FDR = 0.02), cortisol synthesis and secretion (FDR = 0.026), and dopaminergic synapse (FDR = 0.038). Additional enriched pathways included hormone signaling (FDRs < 0.0298, including insulin, GnRH, aldosterone, and growth hormone pathways) and, notably, adrenergic signaling in cardiomyocytes (FDR = 0.0028). These pathways highlight shared molecular mechanisms in the three disorders. Single-nuclei RNA sequencing data from three cortical regions revealed that these core set genes are predominantly expressed in excitatory neuron layers 2–6 of the dorsolateral prefrontal cortex, linking molecular changes to cell types involved in cognitive dysfunction. Our results demonstrate the power of integrating multimodal genetic and transcriptomic data at the exon level. This approach moves beyond symptom-based diagnoses toward molecular classifications, identifying potential therapeutic targets for psychiatric disorders.
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spelling doaj-art-ebb5fe6cab2d4d8a9c9000a8d8339fc32025-08-20T03:18:31ZengNature Publishing GroupTranslational Psychiatry2158-31882025-04-0115111310.1038/s41398-025-03366-8Exon-variant interplay and multi-modal evidence identify endocrine dysregulation in severe psychiatric disorders impacting excitatory neuronsKarolina Worf0Natalie Matosin1Nathalie Gerstner2Anna S. Fröhlich3Anna C. Koller4Franziska Degenhardt5Holger Thiele6Marcella Rietschel7Madhara Udawela8Elizabeth Scarr9Brian Dean10Fabian J. Theis11Nikola S. Mueller12Janine Knauer-Arloth13Institute of Computational Biology, Helmholtz CenterDepartment of Gene and Environment, Max Planck Institute of PsychiatryInstitute of Computational Biology, Helmholtz CenterDepartment of Gene and Environment, Max Planck Institute of PsychiatryInstitute of Human Genetics, University of BonnInstitute of Human Genetics, University of BonnCologne Center for Genomics, University of CologneDepartment of Genetic Epidemiology in Psychiatry, Central Institute of Mental Health, University Medical Center Mannheim/University of HeidelbergThe Molecular Psychiatry Laboratory, The Florey Institute of Neuroscience and Mental HealthThe Department of Psychiatry, The University of MelbourneThe Molecular Psychiatry Laboratory, The Florey Institute of Neuroscience and Mental HealthInstitute of Computational Biology, Helmholtz CenterInstitute of Computational Biology, Helmholtz CenterInstitute of Computational Biology, Helmholtz CenterAbstract Bipolar disorder (BD), major depressive disorder (MDD), and schizophrenia share genetic architecture, yet their molecular mechanisms remain elusive. Both common and rare genetic variants contribute to neural dysfunction, impacting cognition and behavior. This study investigates the molecular effects of genetic variants on human cortical single-cell types using a single-exon analysis approach. Integrating exon-level eQTLs (common variants influencing exon expression) and joint exon eQT-Scores (combining polygenic risk scores with exon-level gene expression) from a postmortem psychiatric cohort (BD = 15, MDD = 24, schizophrenia = 68, controls = 62) with schizophrenia-focused rare variant data from the SCHEMA consortium, we identified 110 core genes enriched in pathways including circadian entrainment (FDR = 0.02), cortisol synthesis and secretion (FDR = 0.026), and dopaminergic synapse (FDR = 0.038). Additional enriched pathways included hormone signaling (FDRs < 0.0298, including insulin, GnRH, aldosterone, and growth hormone pathways) and, notably, adrenergic signaling in cardiomyocytes (FDR = 0.0028). These pathways highlight shared molecular mechanisms in the three disorders. Single-nuclei RNA sequencing data from three cortical regions revealed that these core set genes are predominantly expressed in excitatory neuron layers 2–6 of the dorsolateral prefrontal cortex, linking molecular changes to cell types involved in cognitive dysfunction. Our results demonstrate the power of integrating multimodal genetic and transcriptomic data at the exon level. This approach moves beyond symptom-based diagnoses toward molecular classifications, identifying potential therapeutic targets for psychiatric disorders.https://doi.org/10.1038/s41398-025-03366-8
spellingShingle Karolina Worf
Natalie Matosin
Nathalie Gerstner
Anna S. Fröhlich
Anna C. Koller
Franziska Degenhardt
Holger Thiele
Marcella Rietschel
Madhara Udawela
Elizabeth Scarr
Brian Dean
Fabian J. Theis
Nikola S. Mueller
Janine Knauer-Arloth
Exon-variant interplay and multi-modal evidence identify endocrine dysregulation in severe psychiatric disorders impacting excitatory neurons
Translational Psychiatry
title Exon-variant interplay and multi-modal evidence identify endocrine dysregulation in severe psychiatric disorders impacting excitatory neurons
title_full Exon-variant interplay and multi-modal evidence identify endocrine dysregulation in severe psychiatric disorders impacting excitatory neurons
title_fullStr Exon-variant interplay and multi-modal evidence identify endocrine dysregulation in severe psychiatric disorders impacting excitatory neurons
title_full_unstemmed Exon-variant interplay and multi-modal evidence identify endocrine dysregulation in severe psychiatric disorders impacting excitatory neurons
title_short Exon-variant interplay and multi-modal evidence identify endocrine dysregulation in severe psychiatric disorders impacting excitatory neurons
title_sort exon variant interplay and multi modal evidence identify endocrine dysregulation in severe psychiatric disorders impacting excitatory neurons
url https://doi.org/10.1038/s41398-025-03366-8
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