ArticleMolecular therapy : the journal of the American Society of Gene Therapy2018
Repeated Mesenchymal Stromal Cell Treatment Sustainably Alleviates Machado-Joseph Disease.
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Article in Molecular therapy : the journal of the American Society of Gene Therapy, 2018. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 21 papers.
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21citing papers in PubMed
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21 citing papers in PubMed, 39 citations in OpenAlex.
- Mesenchymal Stem Cell-Based Therapy for Cerebellar Ataxia: From Bench to Bedside.CNS neuroscience & therapeutics · 2026Review
- Post-symptomatic administration of hMSCs exerts therapeutic effects in SCA2 mice.Stem cell research & therapy · 2024Article
- The Potential of Mesenchymal Stem Cells in Treating Spinocerebellar Ataxia: Advances and Future Directions.Biomedicines · 2024Review
- Intracerebellar injection of monocytic immature myeloid cells prevents the adverse effects caused by stereotactic surgery in a model of cerebellar neurodegeneration.Journal of neuroinflammation · 2024Article
- Graft-derived neurons and bystander effects are maintained for six months after human iPSC-derived NESC transplantation in mice's cerebella.Scientific reports · 2024Article
- Editorial: Mesenchymal and induced-pluripotent stem cells as models to study biological processes.Frontiers in genetics · 2024Article
- Cell-based therapeutic strategies for treatment of spinocerebellar ataxias: an update.Neural regeneration research · 2023Review
- A standardised protocol for blood and cerebrospinal fluid collection and processing for biomarker research in ataxia.Neuropathology and applied neurobiology · 2023Article
- Mesenchymal Stem Cell Transplantation Ameliorates Ara-C-Induced Motor Deficits in a Mouse Model of Cerebellar Ataxia.Journal of clinical medicine · 2023Article
- Transcription factor EB-mediated mesenchymal stem cell therapy induces autophagy and alleviates spinocerebellar ataxia type 3 defects in neuronal cells model.Cell death & disease · 2022Article
- Cerebellar morphometric and spectroscopic biomarkers for Machado-Joseph Disease.Acta neuropathologica communications · 2022Article
- ULK overexpression mitigates motor deficits and neuropathology in mouse models of Machado-Joseph disease.Molecular therapy : the journal of the American Society of Gene Therapy · 2022Article
- Preclinical Assessment of Mesenchymal-Stem-Cell-Based Therapies in Spinocerebellar Ataxia Type 3.Biomedicines · 2021Article
- Mutant Ataxin-3-Containing Aggregates (MATAGGs) in Spinocerebellar Ataxia Type 3: Dynamics of the Disorder.Molecular neurobiology · 2021Review
- Current Status of Gene Therapy Research in Polyglutamine Spinocerebellar Ataxias.International journal of molecular sciences · 2021Review
- The blood-brain barrier is disrupted in Machado-Joseph disease/spinocerebellar ataxia type 3: evidence from transgenic mice and human post-mortem samples.Acta neuropathologica communications · 2020Article
- Mesenchymal stem cell-derived exosomes improve motor function and attenuate neuropathology in a mouse model of Machado-Joseph disease.Stem cell research & therapy · 2020Article
- The neurological update: therapies for cerebellar ataxias in 2020.Journal of neurology · 2020Review
- Mesenchymal Stromal Cells' Therapy for Polyglutamine Disorders: Where Do We Stand and Where Should We Go?Frontiers in cellular neuroscience · 2020Review
- Intraportally delivered stem cell spheroids localize in the liver and protect hepatocytes against GalN/LPS-induced fulminant hepatic toxicity.Stem cell research & therapy · 2019Article
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Authors and funding
21 authors at 1 institution in 1 country.
Catarina Oliveira MirandaCenter for Neuroscience and Cell Biology (CNC), University of Coimbra, Faculdade de Medicina, Rua Larga, Pólo I, 1° andar, 3004-504 Coimbra, Portugal; Institute for Interdisciplinary Research, University of Coimbra, Casa Costa Alemão - Pólo II, Rua Dom Francisco de Lemos, 3030-789 Coimbra, Portugal; Centre for Neuroscience and Cell Biology - Institute of Biomedical Imaging and Life Science (CNC.IBILI), University of Coimbra, Azinhaga de Santa Comba, 3000-548 Coimbra, Portugal.
Adriana MarceloCenter for Neuroscience and Cell Biology (CNC), University of Coimbra, Faculdade de Medicina, Rua Larga, Pólo I, 1° andar, 3004-504 Coimbra, Portugal; Centre for Neuroscience and Cell Biology - Institute of Biomedical Imaging and Life Science (CNC.IBILI), University of Coimbra, Azinhaga de Santa Comba, 3000-548 Coimbra, Portugal.
Teresa Pereira SilvaCenter for Neuroscience and Cell Biology (CNC), University of Coimbra, Faculdade de Medicina, Rua Larga, Pólo I, 1° andar, 3004-504 Coimbra, Portugal; Centre for Neuroscience and Cell Biology - Institute of Biomedical Imaging and Life Science (CNC.IBILI), University of Coimbra, Azinhaga de Santa Comba, 3000-548 Coimbra, Portugal.
João BarataCenter for Neuroscience and Cell Biology (CNC), University of Coimbra, Faculdade de Medicina, Rua Larga, Pólo I, 1° andar, 3004-504 Coimbra, Portugal; Centre for Neuroscience and Cell Biology - Institute of Biomedical Imaging and Life Science (CNC.IBILI), University of Coimbra, Azinhaga de Santa Comba, 3000-548 Coimbra, Portugal.
Ana Vasconcelos-FerreiraCenter for Neuroscience and Cell Biology (CNC), University of Coimbra, Faculdade de Medicina, Rua Larga, Pólo I, 1° andar, 3004-504 Coimbra, Portugal; Institute for Interdisciplinary Research, University of Coimbra, Casa Costa Alemão - Pólo II, Rua Dom Francisco de Lemos, 3030-789 Coimbra, Portugal; Centre for Neuroscience and Cell Biology - Institute of Biomedical Imaging and Life Science (CNC.IBILI), University of Coimbra, Azinhaga de Santa Comba, 3000-548 Coimbra, Portugal; Doctoral Programme of Faculty of Pharmacy, University of Coimbra, 3000-548 Coimbra, Portugal.
Dina PereiraCenter for Neuroscience and Cell Biology (CNC), University of Coimbra, Faculdade de Medicina, Rua Larga, Pólo I, 1° andar, 3004-504 Coimbra, Portugal; Institute for Interdisciplinary Research, University of Coimbra, Casa Costa Alemão - Pólo II, Rua Dom Francisco de Lemos, 3030-789 Coimbra, Portugal; Centre for Neuroscience and Cell Biology - Institute of Biomedical Imaging and Life Science (CNC.IBILI), University of Coimbra, Azinhaga de Santa Comba, 3000-548 Coimbra, Portugal; Doctoral Programme in Experimental Biology and Biomedicine, CNC - University of Coimbra, Rua Larga, Faculdade de Medicina, Pólo I, 1° andar, 3004-504 Coimbra, Portugal.
Clévio NóbregaCenter for Neuroscience and Cell Biology (CNC), University of Coimbra, Faculdade de Medicina, Rua Larga, Pólo I, 1° andar, 3004-504 Coimbra, Portugal; Institute for Interdisciplinary Research, University of Coimbra, Casa Costa Alemão - Pólo II, Rua Dom Francisco de Lemos, 3030-789 Coimbra, Portugal; Centre for Neuroscience and Cell Biology - Institute of Biomedical Imaging and Life Science (CNC.IBILI), University of Coimbra, Azinhaga de Santa Comba, 3000-548 Coimbra, Portugal.
Sónia DuarteCenter for Neuroscience and Cell Biology (CNC), University of Coimbra, Faculdade de Medicina, Rua Larga, Pólo I, 1° andar, 3004-504 Coimbra, Portugal; Institute for Interdisciplinary Research, University of Coimbra, Casa Costa Alemão - Pólo II, Rua Dom Francisco de Lemos, 3030-789 Coimbra, Portugal; Centre for Neuroscience and Cell Biology - Institute of Biomedical Imaging and Life Science (CNC.IBILI), University of Coimbra, Azinhaga de Santa Comba, 3000-548 Coimbra, Portugal.
Inês BarrosCenter for Neuroscience and Cell Biology (CNC), University of Coimbra, Faculdade de Medicina, Rua Larga, Pólo I, 1° andar, 3004-504 Coimbra, Portugal; Centre for Neuroscience and Cell Biology - Institute of Biomedical Imaging and Life Science (CNC.IBILI), University of Coimbra, Azinhaga de Santa Comba, 3000-548 Coimbra, Portugal.
Joana AlvesCenter for Neuroscience and Cell Biology (CNC), University of Coimbra, Faculdade de Medicina, Rua Larga, Pólo I, 1° andar, 3004-504 Coimbra, Portugal; Centre for Neuroscience and Cell Biology - Institute of Biomedical Imaging and Life Science (CNC.IBILI), University of Coimbra, Azinhaga de Santa Comba, 3000-548 Coimbra, Portugal.
José SerenoCentre for Neuroscience and Cell Biology - Institute of Biomedical Imaging and Life Science (CNC.IBILI), University of Coimbra, Azinhaga de Santa Comba, 3000-548 Coimbra, Portugal; Coimbra Institute for Biomedical Imaging and Translational Research, Edifício do ICNAS, Polo 3, Azinhaga de Santa Comba, 3000-548 Coimbra, Portugal; Institute of Nuclear Science Applied to Health, University of Coimbra, Polo 3, Azinhaga de Santa Comba, 3000-548 Coimbra, Portugal.
Lorena Itatí PetrellaCentre for Neuroscience and Cell Biology - Institute of Biomedical Imaging and Life Science (CNC.IBILI), University of Coimbra, Azinhaga de Santa Comba, 3000-548 Coimbra, Portugal; Coimbra Institute for Biomedical Imaging and Translational Research, Edifício do ICNAS, Polo 3, Azinhaga de Santa Comba, 3000-548 Coimbra, Portugal; Institute of Nuclear Science Applied to Health, University of Coimbra, Polo 3, Azinhaga de Santa Comba, 3000-548 Coimbra, Portugal.
João CastelhanoCentre for Neuroscience and Cell Biology - Institute of Biomedical Imaging and Life Science (CNC.IBILI), University of Coimbra, Azinhaga de Santa Comba, 3000-548 Coimbra, Portugal; Coimbra Institute for Biomedical Imaging and Translational Research, Edifício do ICNAS, Polo 3, Azinhaga de Santa Comba, 3000-548 Coimbra, Portugal; Institute of Nuclear Science Applied to Health, University of Coimbra, Polo 3, Azinhaga de Santa Comba, 3000-548 Coimbra, Portugal.
Vitor Hugo PaivaInstitute for Interdisciplinary Research, University of Coimbra, Casa Costa Alemão - Pólo II, Rua Dom Francisco de Lemos, 3030-789 Coimbra, Portugal; MARE - Marine and Environmental Sciences Centre, Department of Life Sciences, University of Coimbra, 3004-517 Coimbra, Portugal.
Paulo Rodrigues-SantosImmunology Institute, Faculty of Medicine, University of Coimbra, Rua Larga, 3004-504 Coimbra, Portugal; Immunology and Oncology Laboratory, Center for Neurosciences and Cell Biology (CNC), University of Coimbra, Rua Larga, 3004-504, Portugal; Center of Investigation in Environment, Genetics and Oncobiology, Apartado 9015, 3001-301, Coimbra, Portugal.
Vera AlvesImmunology Institute, Faculty of Medicine, University of Coimbra, Rua Larga, 3004-504 Coimbra, Portugal; Immunology and Oncology Laboratory, Center for Neurosciences and Cell Biology (CNC), University of Coimbra, Rua Larga, 3004-504, Portugal; Center of Investigation in Environment, Genetics and Oncobiology, Apartado 9015, 3001-301, Coimbra, Portugal.
Isabel Nunes-CorreiaCenter for Neuroscience and Cell Biology (CNC), University of Coimbra, Faculdade de Medicina, Rua Larga, Pólo I, 1° andar, 3004-504 Coimbra, Portugal; Centre for Neuroscience and Cell Biology - Institute of Biomedical Imaging and Life Science (CNC.IBILI), University of Coimbra, Azinhaga de Santa Comba, 3000-548 Coimbra, Portugal.
Rui Jorge NobreCenter for Neuroscience and Cell Biology (CNC), University of Coimbra, Faculdade de Medicina, Rua Larga, Pólo I, 1° andar, 3004-504 Coimbra, Portugal; Institute for Interdisciplinary Research, University of Coimbra, Casa Costa Alemão - Pólo II, Rua Dom Francisco de Lemos, 3030-789 Coimbra, Portugal; Centre for Neuroscience and Cell Biology - Institute of Biomedical Imaging and Life Science (CNC.IBILI), University of Coimbra, Azinhaga de Santa Comba, 3000-548 Coimbra, Portugal.
Célia GomesCoimbra Institute for Clinical and Biomedical Research, Faculty of Medicine, University of Coimbra, Polo 3, Azinhaga de Santa Comba, 3000-548 Coimbra, Portugal.
Miguel Castelo-BrancoCentre for Neuroscience and Cell Biology - Institute of Biomedical Imaging and Life Science (CNC.IBILI), University of Coimbra, Azinhaga de Santa Comba, 3000-548 Coimbra, Portugal; Coimbra Institute for Biomedical Imaging and Translational Research, Edifício do ICNAS, Polo 3, Azinhaga de Santa Comba, 3000-548 Coimbra, Portugal; Institute of Nuclear Science Applied to Health, University of Coimbra, Polo 3, Azinhaga de Santa Comba, 3000-548 Coimbra, Portugal.
Luís Pereira de AlmeidaCenter for Neuroscience and Cell Biology (CNC), University of Coimbra, Faculdade de Medicina, Rua Larga, Pólo I, 1° andar, 3004-504 Coimbra, Portugal; Faculty of Pharmacy, University of Coimbra, Polo 3, Azinhaga de Santa Comba, 3000-548 Coimbra, Portugal. Electronic address: luispa@cnc.uc.pt.
University of Coimbra · PT
Funding
No grant is acknowledged in the PubMed record.
Abstract
Machado-Joseph disease (MJD) or spinocerebellar ataxia type 3, the most common dominant spinocerebellar ataxia (SCA) worldwide, is caused by over-repetition of a CAG repeat in the ATXN3/MJD1 gene, which translates into a polyglutamine tract within the ataxin-3 protein. There is no treatment for this fatal disorder. Despite evidence of the safety and efficacy of mesenchymal stromal cells (MSCs) in delaying SCA disease progression in exploratory clinical trials, unanticipated regression of patients to the status prior to treatment makes the investigation of causes and solutions urgent and imperative. In the present study, we compared the efficacy of a single intracranial injection with repeated systemic MSC administration in alleviating the MJD phenotype of two strongly severe genetic rodent models. We found that a single MSC transplantation only produces transient effects, whereas periodic administration promotes sustained motor behavior and neuropathology alleviation, suggesting that MSC therapies should be re-designed to get sustained beneficial results in clinical practice. Furthermore, MSC promoted neuroprotection, increased the levels of GABA and glutamate, and decreased the levels of Myo-inositol, which correlated with motor improvements, indicating that these metabolites may serve as valid neurospectroscopic biomarkers of disease and treatment. This study makes important contributions to the design of new clinical approaches for MJD and other SCAs/polyglutamine disorders.
Indexed as
AnimalsAtaxin-3gamma-Aminobutyric AcidGlutamic AcidMachado-Joseph DiseaseMaleMesenchymal Stem CellsMesenchymal Stem Cell TransplantationMiceMice, Inbred C57BLMice, TransgenicAtaxin-3Atxn3 protein, mousegamma-Aminobutyric AcidGlutamic Acid1H-MRSdisease and treatment markers GABA and glutamateintravenous repeated treatmentin vivo proton magnetic resonance spectroscopyMachado-Joseph diseasemesenchymal stromal (stem) cellsMJDmouse modelsneuroprotectionSCA-3short lifetime after in vivo transplantationspinocerebellar ataxia type 3sustainable motor improvements
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