Efficacy of human umbilical cord mesenchymal stem cell in the treatment of neuromyelitis optica spectrum disorders: an animal study

Abstract Background Human umbilical cord mesenchymal stem cells (hUC-MSCs) have great potential for treating autoimmune diseases for their immunomodulatory and tissue-regenerative abilities; however, their therapeutic role in neuromyelitis optica spectrum disorder (NMOSD) remains uncertain. Methods...

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Main Authors: Chunran Xue, Haojun Yu, Xuzhong Pei, Xiaoying Yao, Jie Ding, Xiying Wang, Yi Chen, Yangtai Guan
Format: Article
Language:English
Published: BMC 2025-02-01
Series:Stem Cell Research & Therapy
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Online Access:https://doi.org/10.1186/s13287-025-04187-8
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author Chunran Xue
Haojun Yu
Xuzhong Pei
Xiaoying Yao
Jie Ding
Xiying Wang
Yi Chen
Yangtai Guan
author_facet Chunran Xue
Haojun Yu
Xuzhong Pei
Xiaoying Yao
Jie Ding
Xiying Wang
Yi Chen
Yangtai Guan
author_sort Chunran Xue
collection DOAJ
description Abstract Background Human umbilical cord mesenchymal stem cells (hUC-MSCs) have great potential for treating autoimmune diseases for their immunomodulatory and tissue-regenerative abilities; however, their therapeutic role in neuromyelitis optica spectrum disorder (NMOSD) remains uncertain. Methods 106 hUC-MSCs prepared in 200 μl PBS were intravenously administered to a systemic NMOSD model on day 10 and day 14 after immunization. Then, disease progression, immune responses, and blood–brain barrier integrity were evaluated. Additionally, we tested the effects of hUC-MSCs on astrocyte viability and apoptosis using an aquaporin 4 (AQP4) IgG and complement-induced cytotoxicity model in vitro. Results hUC-MSCs alleviated NMOSD progression in vivo with improved motor function, reduced inflammatory infiltration, myelin loss, and preservation of astrocytes and neurons. hUC-MSC treatment did not affect autoimmune reactions in the spleen, however, decreased cytokine release in the spinal cord and mitigated blood–brain barrier disruption. Furthermore, in vitro studies revealed that co-culture with hUC-MSCs significantly restored astrocyte viability and reduced apoptosis in AQP4 IgG and complement-mediated damage. Conclusion Our results revealed that hUC-MSCs displayed therapeutic efficacy in NMOSD and showed potential in attenuating blood–brain barrier disruption, as well as AQP4 IgG and complement-induced astrocyte apoptosis.
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publishDate 2025-02-01
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spelling doaj-art-4c01a905e6724295a0bb11d8fa97c0e82025-02-09T12:15:43ZengBMCStem Cell Research & Therapy1757-65122025-02-0116111310.1186/s13287-025-04187-8Efficacy of human umbilical cord mesenchymal stem cell in the treatment of neuromyelitis optica spectrum disorders: an animal studyChunran Xue0Haojun Yu1Xuzhong Pei2Xiaoying Yao3Jie Ding4Xiying Wang5Yi Chen6Yangtai Guan7Department of Neurology, Renji Hospital, Shanghai Jiaotong University School of MedicineDepartment of Neurology, Renji Hospital, Shanghai Jiaotong University School of MedicineSchool of Medicine, Shanghai UniversityDepartment of Neurology, Renji Hospital, Shanghai Jiaotong University School of MedicineDepartment of Neurology, Renji Hospital, Shanghai Jiaotong University School of MedicineDepartment of Neurology, Renji Hospital, Shanghai Jiaotong University School of MedicineDepartment of Neurology, Renji Hospital, Shanghai Jiaotong University School of MedicineDepartment of Neurology, Punan Branch of Renji Hospital, Shanghai Jiaotong University School of Medicine (Punan Hospital in Pudong New District, Shanghai)Abstract Background Human umbilical cord mesenchymal stem cells (hUC-MSCs) have great potential for treating autoimmune diseases for their immunomodulatory and tissue-regenerative abilities; however, their therapeutic role in neuromyelitis optica spectrum disorder (NMOSD) remains uncertain. Methods 106 hUC-MSCs prepared in 200 μl PBS were intravenously administered to a systemic NMOSD model on day 10 and day 14 after immunization. Then, disease progression, immune responses, and blood–brain barrier integrity were evaluated. Additionally, we tested the effects of hUC-MSCs on astrocyte viability and apoptosis using an aquaporin 4 (AQP4) IgG and complement-induced cytotoxicity model in vitro. Results hUC-MSCs alleviated NMOSD progression in vivo with improved motor function, reduced inflammatory infiltration, myelin loss, and preservation of astrocytes and neurons. hUC-MSC treatment did not affect autoimmune reactions in the spleen, however, decreased cytokine release in the spinal cord and mitigated blood–brain barrier disruption. Furthermore, in vitro studies revealed that co-culture with hUC-MSCs significantly restored astrocyte viability and reduced apoptosis in AQP4 IgG and complement-mediated damage. Conclusion Our results revealed that hUC-MSCs displayed therapeutic efficacy in NMOSD and showed potential in attenuating blood–brain barrier disruption, as well as AQP4 IgG and complement-induced astrocyte apoptosis.https://doi.org/10.1186/s13287-025-04187-8Aquaporin 4AstrocyteMesenchymal stem cellsNeuromyelitis optica spectrum disorderUmbilical cord
spellingShingle Chunran Xue
Haojun Yu
Xuzhong Pei
Xiaoying Yao
Jie Ding
Xiying Wang
Yi Chen
Yangtai Guan
Efficacy of human umbilical cord mesenchymal stem cell in the treatment of neuromyelitis optica spectrum disorders: an animal study
Stem Cell Research & Therapy
Aquaporin 4
Astrocyte
Mesenchymal stem cells
Neuromyelitis optica spectrum disorder
Umbilical cord
title Efficacy of human umbilical cord mesenchymal stem cell in the treatment of neuromyelitis optica spectrum disorders: an animal study
title_full Efficacy of human umbilical cord mesenchymal stem cell in the treatment of neuromyelitis optica spectrum disorders: an animal study
title_fullStr Efficacy of human umbilical cord mesenchymal stem cell in the treatment of neuromyelitis optica spectrum disorders: an animal study
title_full_unstemmed Efficacy of human umbilical cord mesenchymal stem cell in the treatment of neuromyelitis optica spectrum disorders: an animal study
title_short Efficacy of human umbilical cord mesenchymal stem cell in the treatment of neuromyelitis optica spectrum disorders: an animal study
title_sort efficacy of human umbilical cord mesenchymal stem cell in the treatment of neuromyelitis optica spectrum disorders an animal study
topic Aquaporin 4
Astrocyte
Mesenchymal stem cells
Neuromyelitis optica spectrum disorder
Umbilical cord
url https://doi.org/10.1186/s13287-025-04187-8
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