PBMC-Humanized Mouse Model for Multiple Sclerosis: Studying Immune Changes and CNS Involvement

Humanized immune system (HIS) mice are powerful tools for studying human immune system function and dysfunction and developing human-specific immunotherapeutics. The availability of sophisticated super immunodeficient mouse strains has allowed immune system humanization using transplants of human pe...

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Main Authors: Anastasia Dagkonaki, Irini Papazian, Vasileios Gouzouasis, Ariadni Karles, Maria Kourouvani, Theodore Tselios, Eirini Fragkiadaki, Lesley Probert
Format: Article
Language:English
Published: Bio-protocol LLC 2025-05-01
Series:Bio-Protocol
Online Access:https://bio-protocol.org/en/bpdetail?id=5312&type=0
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author Anastasia Dagkonaki
Irini Papazian
Vasileios Gouzouasis
Ariadni Karles
Maria Kourouvani
Theodore Tselios
Eirini Fragkiadaki
Lesley Probert
author_facet Anastasia Dagkonaki
Irini Papazian
Vasileios Gouzouasis
Ariadni Karles
Maria Kourouvani
Theodore Tselios
Eirini Fragkiadaki
Lesley Probert
author_sort Anastasia Dagkonaki
collection DOAJ
description Humanized immune system (HIS) mice are powerful tools for studying human immune system function and dysfunction and developing human-specific immunotherapeutics. The availability of sophisticated super immunodeficient mouse strains has allowed immune system humanization using transplants of human peripheral blood mononuclear cells (PBMC) or hematopoietic stem cells. HIS mice are used extensively in immune-oncology, while there are fewer studies in autoimmunity, especially multiple sclerosis (MS). Using the protocol described here, we generated HIS mice that show key features of MS not represented in other widely used MS models [1]. Severely immunodeficient NOD.Cg-B2mem1Tac Prkdcscid Il2rgtm1Sug/JicTac (B2m-NOG) mice, which lack murine B, T, and NK cells and murine major histocompatibility class I molecules and have defective innate immune responses, were transplanted with PBMC from HLA-DRB1-genotyped MS patients and healthy donors. Mice were successfully engrafted with hCD4 and hCD8 T and B lymphocytes and developed both spontaneous and experimental autoimmune encephalomyelitis (EAE)-enhanced T-cell lesions in the central nervous system. B-cell engraftment was highest in mice receiving cells from MS patients with serological evidence for Epstein–Barr virus (EBV) reactivation. This humanized MS model shows advantages over EAE, particularly spontaneous hCD8 T-cell lesions in the brain and spinal cord, mixed hCD8/hCD4 T-cell lesions in EAE-immunized mice, and more severe lesions in mice engrafted with PBMC from MS donors carrying the DRB1*15 MS susceptibility allele compared to DRB1*15-positive healthy and DRB1*13-positive MS donors. MS HIS mice represent simple and rapid tools for investigating human immunopathology and the efficacy of therapeutics at a personalized level.
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spelling doaj-art-4a8cf3c6faa042678f033e9699e1a14d2025-08-20T03:21:32ZengBio-protocol LLCBio-Protocol2331-83252025-05-01151010.21769/BioProtoc.5312PBMC-Humanized Mouse Model for Multiple Sclerosis: Studying Immune Changes and CNS InvolvementAnastasia Dagkonaki0Irini Papazian1Vasileios Gouzouasis2Ariadni Karles3Maria Kourouvani4Theodore Tselios5Eirini Fragkiadaki6Lesley Probert7Laboratory of Molecular Genetics, Department of Immunology, Hellenic Pasteur Institute, Athens, GreeceLaboratory of Molecular Genetics, Department of Immunology, Hellenic Pasteur Institute, Athens, GreeceLaboratory of Molecular Genetics, Department of Immunology, Hellenic Pasteur Institute, Athens, GreeceDepartment of Animal Models for Biomedical Research, Hellenic Pasteur Institute, Athens, GreeceLaboratory of Molecular Genetics, Department of Immunology, Hellenic Pasteur Institute, Athens, GreeceDepartment of Chemistry, University of Patras, Patras, GreeceDepartment of Animal Models for Biomedical Research, Hellenic Pasteur Institute, Athens, GreeceLaboratory of Molecular Genetics, Department of Immunology, Hellenic Pasteur Institute, Athens, GreeceHumanized immune system (HIS) mice are powerful tools for studying human immune system function and dysfunction and developing human-specific immunotherapeutics. The availability of sophisticated super immunodeficient mouse strains has allowed immune system humanization using transplants of human peripheral blood mononuclear cells (PBMC) or hematopoietic stem cells. HIS mice are used extensively in immune-oncology, while there are fewer studies in autoimmunity, especially multiple sclerosis (MS). Using the protocol described here, we generated HIS mice that show key features of MS not represented in other widely used MS models [1]. Severely immunodeficient NOD.Cg-B2mem1Tac Prkdcscid Il2rgtm1Sug/JicTac (B2m-NOG) mice, which lack murine B, T, and NK cells and murine major histocompatibility class I molecules and have defective innate immune responses, were transplanted with PBMC from HLA-DRB1-genotyped MS patients and healthy donors. Mice were successfully engrafted with hCD4 and hCD8 T and B lymphocytes and developed both spontaneous and experimental autoimmune encephalomyelitis (EAE)-enhanced T-cell lesions in the central nervous system. B-cell engraftment was highest in mice receiving cells from MS patients with serological evidence for Epstein–Barr virus (EBV) reactivation. This humanized MS model shows advantages over EAE, particularly spontaneous hCD8 T-cell lesions in the brain and spinal cord, mixed hCD8/hCD4 T-cell lesions in EAE-immunized mice, and more severe lesions in mice engrafted with PBMC from MS donors carrying the DRB1*15 MS susceptibility allele compared to DRB1*15-positive healthy and DRB1*13-positive MS donors. MS HIS mice represent simple and rapid tools for investigating human immunopathology and the efficacy of therapeutics at a personalized level.https://bio-protocol.org/en/bpdetail?id=5312&type=0
spellingShingle Anastasia Dagkonaki
Irini Papazian
Vasileios Gouzouasis
Ariadni Karles
Maria Kourouvani
Theodore Tselios
Eirini Fragkiadaki
Lesley Probert
PBMC-Humanized Mouse Model for Multiple Sclerosis: Studying Immune Changes and CNS Involvement
Bio-Protocol
title PBMC-Humanized Mouse Model for Multiple Sclerosis: Studying Immune Changes and CNS Involvement
title_full PBMC-Humanized Mouse Model for Multiple Sclerosis: Studying Immune Changes and CNS Involvement
title_fullStr PBMC-Humanized Mouse Model for Multiple Sclerosis: Studying Immune Changes and CNS Involvement
title_full_unstemmed PBMC-Humanized Mouse Model for Multiple Sclerosis: Studying Immune Changes and CNS Involvement
title_short PBMC-Humanized Mouse Model for Multiple Sclerosis: Studying Immune Changes and CNS Involvement
title_sort pbmc humanized mouse model for multiple sclerosis studying immune changes and cns involvement
url https://bio-protocol.org/en/bpdetail?id=5312&type=0
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