A microglia clonal inflammatory disorder in Alzheimer’s disease

Somatic genetic heterogeneity resulting from post-zygotic DNA mutations is widespread in human tissues and can cause diseases, however, few studies have investigated its role in neurodegenerative processes such as Alzheimer’s disease (AD). Here, we report the selective enrichment of microglia clones...

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Main Authors: Rocio Vicario, Stamatina Fragkogianni, Leslie Weber, Tomi Lazarov, Yang Hu, Samantha Y Hayashi, Barbara Craddock, Nicholas D Socci, Araitz Alberdi, Ann Baako, Oyku Ay, Masato Ogishi, Estibaliz Lopez-Rodrigo, Rajya Kappagantula, Agnes Viale, Christine A Iacobuzio-Donahue, Ting Zhou, Richard M Ransohoff, Richard Chesworth, Netherlands Brain Bank, Omar Abdel-Wahab, Bertrand Boisson, Olivier Elemento, Jean-Laurent Casanova, W Todd Miller, Frédéric Geissmann
Format: Article
Language:English
Published: eLife Sciences Publications Ltd 2025-03-01
Series:eLife
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Online Access:https://elifesciences.org/articles/96519
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author Rocio Vicario
Stamatina Fragkogianni
Leslie Weber
Tomi Lazarov
Yang Hu
Samantha Y Hayashi
Barbara Craddock
Nicholas D Socci
Araitz Alberdi
Ann Baako
Oyku Ay
Masato Ogishi
Estibaliz Lopez-Rodrigo
Rajya Kappagantula
Agnes Viale
Christine A Iacobuzio-Donahue
Ting Zhou
Richard M Ransohoff
Richard Chesworth
Netherlands Brain Bank
Omar Abdel-Wahab
Bertrand Boisson
Olivier Elemento
Jean-Laurent Casanova
W Todd Miller
Frédéric Geissmann
author_facet Rocio Vicario
Stamatina Fragkogianni
Leslie Weber
Tomi Lazarov
Yang Hu
Samantha Y Hayashi
Barbara Craddock
Nicholas D Socci
Araitz Alberdi
Ann Baako
Oyku Ay
Masato Ogishi
Estibaliz Lopez-Rodrigo
Rajya Kappagantula
Agnes Viale
Christine A Iacobuzio-Donahue
Ting Zhou
Richard M Ransohoff
Richard Chesworth
Netherlands Brain Bank
Omar Abdel-Wahab
Bertrand Boisson
Olivier Elemento
Jean-Laurent Casanova
W Todd Miller
Frédéric Geissmann
author_sort Rocio Vicario
collection DOAJ
description Somatic genetic heterogeneity resulting from post-zygotic DNA mutations is widespread in human tissues and can cause diseases, however, few studies have investigated its role in neurodegenerative processes such as Alzheimer’s disease (AD). Here, we report the selective enrichment of microglia clones carrying pathogenic variants, that are not present in neuronal, glia/stromal cells, or blood, from patients with AD in comparison to age-matched controls. Notably, microglia-specific AD-associated variants preferentially target the MAPK pathway, including recurrent CBL ring-domain mutations. These variants activate ERK and drive a microglia transcriptional program characterized by a strong neuro-inflammatory response, both in vitro and in patients. Although the natural history of AD-associated microglial clones is difficult to establish in humans, microglial expression of a MAPK pathway activating variant was previously shown to cause neurodegeneration in mice, suggesting that AD-associated neuroinflammatory microglial clones may contribute to the neurodegenerative process in patients.
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spelling doaj-art-b7b9309b8c854c5ba616612ffa5b7fe42025-08-20T02:57:04ZengeLife Sciences Publications LtdeLife2050-084X2025-03-011310.7554/eLife.96519A microglia clonal inflammatory disorder in Alzheimer’s diseaseRocio Vicario0https://orcid.org/0000-0002-7894-5261Stamatina Fragkogianni1Leslie Weber2Tomi Lazarov3https://orcid.org/0000-0002-6312-0080Yang Hu4Samantha Y Hayashi5Barbara Craddock6Nicholas D Socci7Araitz Alberdi8Ann Baako9Oyku Ay10Masato Ogishi11Estibaliz Lopez-Rodrigo12Rajya Kappagantula13Agnes Viale14Christine A Iacobuzio-Donahue15Ting Zhou16Richard M Ransohoff17https://orcid.org/0000-0003-0175-6910Richard Chesworth18Netherlands Brain Bank19Omar Abdel-Wahab20https://orcid.org/0000-0002-3907-6171Bertrand Boisson21Olivier Elemento22Jean-Laurent Casanova23W Todd Miller24Frédéric Geissmann25https://orcid.org/0000-0001-5029-2468Immunology Program, Sloan Kettering Institute, Memorial Sloan Kettering Cancer Center, New York, New York, United StatesImmunology Program, Sloan Kettering Institute, Memorial Sloan Kettering Cancer Center, New York, New York, United StatesImmunology Program, Sloan Kettering Institute, Memorial Sloan Kettering Cancer Center, New York, New York, United StatesImmunology Program, Sloan Kettering Institute, Memorial Sloan Kettering Cancer Center, New York, New York, United StatesDepartment of Physiology and Biophysics, Institute for Computational Biomedicine, Weill Cornell New York, New York, United StatesDepartment of Physiology and Biophysics, Stony Brook University School of Medicine, Stony Brook, New York, United StatesDepartment of Physiology and Biophysics, Stony Brook University School of Medicine, Stony Brook, New York, United StatesMarie-Josée & Henry R. Kravis Center for Molecular Oncology, Memorial Sloan Kettering Cancer Center, New York, New York, United StatesImmunology Program, Sloan Kettering Institute, Memorial Sloan Kettering Cancer Center, New York, New York, United StatesImmunology Program, Sloan Kettering Institute, Memorial Sloan Kettering Cancer Center, New York, New York, United StatesImmunology Program, Sloan Kettering Institute, Memorial Sloan Kettering Cancer Center, New York, New York, United StatesSt. Giles Laboratory of Human Genetics of Infectious Diseases, Rockefeller Branch, The Rockefeller University, New York, New York, United StatesImmunology Program, Sloan Kettering Institute, Memorial Sloan Kettering Cancer Center, New York, New York, United StatesHuman Oncology & Pathogenesis Program, Memorial Sloan Kettering Cancer Center, New York, New York, United StatesMarie-Josée & Henry R. Kravis Center for Molecular Oncology, Memorial Sloan Kettering Cancer Center, New York, New York, United StatesHuman Oncology & Pathogenesis Program, Memorial Sloan Kettering Cancer Center, New York, New York, United States; Department of Pathology, Memorial Sloan Kettering Cancer Center, New York, New York, United StatesSKI Stem Cell Research Core, Memorial Sloan Kettering Cancer Center, New York, New York, United StatesThird Rock Ventures, Boston, United StatesThird Rock Ventures, Boston, United StatesNetherlands Brain Bank, Amsterdam, NetherlandsHuman Oncology & Pathogenesis Program, Memorial Sloan Kettering Cancer Center, New York, New York, United StatesSt. Giles Laboratory of Human Genetics of Infectious Diseases, Rockefeller Branch, The Rockefeller University, New York, New York, United StatesDepartment of Physiology and Biophysics, Institute for Computational Biomedicine, Weill Cornell New York, New York, United StatesSt. Giles Laboratory of Human Genetics of Infectious Diseases, Rockefeller Branch, The Rockefeller University, New York, New York, United StatesDepartment of Physiology and Biophysics, Stony Brook University School of Medicine, Stony Brook, New York, United StatesImmunology Program, Sloan Kettering Institute, Memorial Sloan Kettering Cancer Center, New York, New York, United StatesSomatic genetic heterogeneity resulting from post-zygotic DNA mutations is widespread in human tissues and can cause diseases, however, few studies have investigated its role in neurodegenerative processes such as Alzheimer’s disease (AD). Here, we report the selective enrichment of microglia clones carrying pathogenic variants, that are not present in neuronal, glia/stromal cells, or blood, from patients with AD in comparison to age-matched controls. Notably, microglia-specific AD-associated variants preferentially target the MAPK pathway, including recurrent CBL ring-domain mutations. These variants activate ERK and drive a microglia transcriptional program characterized by a strong neuro-inflammatory response, both in vitro and in patients. Although the natural history of AD-associated microglial clones is difficult to establish in humans, microglial expression of a MAPK pathway activating variant was previously shown to cause neurodegeneration in mice, suggesting that AD-associated neuroinflammatory microglial clones may contribute to the neurodegenerative process in patients.https://elifesciences.org/articles/96519somatic mutationsAlzheimer's diseasemicrogliamap kinase
spellingShingle Rocio Vicario
Stamatina Fragkogianni
Leslie Weber
Tomi Lazarov
Yang Hu
Samantha Y Hayashi
Barbara Craddock
Nicholas D Socci
Araitz Alberdi
Ann Baako
Oyku Ay
Masato Ogishi
Estibaliz Lopez-Rodrigo
Rajya Kappagantula
Agnes Viale
Christine A Iacobuzio-Donahue
Ting Zhou
Richard M Ransohoff
Richard Chesworth
Netherlands Brain Bank
Omar Abdel-Wahab
Bertrand Boisson
Olivier Elemento
Jean-Laurent Casanova
W Todd Miller
Frédéric Geissmann
A microglia clonal inflammatory disorder in Alzheimer’s disease
eLife
somatic mutations
Alzheimer's disease
microglia
map kinase
title A microglia clonal inflammatory disorder in Alzheimer’s disease
title_full A microglia clonal inflammatory disorder in Alzheimer’s disease
title_fullStr A microglia clonal inflammatory disorder in Alzheimer’s disease
title_full_unstemmed A microglia clonal inflammatory disorder in Alzheimer’s disease
title_short A microglia clonal inflammatory disorder in Alzheimer’s disease
title_sort microglia clonal inflammatory disorder in alzheimer s disease
topic somatic mutations
Alzheimer's disease
microglia
map kinase
url https://elifesciences.org/articles/96519
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