Extracellular vesicles-associated AAVs for the treatment of Machado-Joseph disease.

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Title: Extracellular vesicles-associated AAVs for the treatment of Machado-Joseph disease.
Authors: Henriques C; Gene and Stem Cell Therapies for the Brain Group, Center for Neuroscience and Cell Biology, University of Coimbra (CNC-UC), 3004-504 Coimbra, Portugal; Center for Innovative Biomedicine and Biotechnology (CIBB), Vectors, Gene and Cell Therapy Group, University of Coimbra, 3004-504 Coimbra, Portugal; GeneT, Center for Excellence in Gene Therapy in Portugal, University of Coimbra, 3004-504 Coimbra, Portugal; ViraVector-Viral Vectors for Gene Transfer Core Facility, University of Coimbra, 3004-504 Coimbra, Portugal; Faculty of Pharmacy, University of Coimbra, 3000-548 Coimbra, Portugal., Albuquerque P; Gene and Stem Cell Therapies for the Brain Group, Center for Neuroscience and Cell Biology, University of Coimbra (CNC-UC), 3004-504 Coimbra, Portugal; Center for Innovative Biomedicine and Biotechnology (CIBB), Vectors, Gene and Cell Therapy Group, University of Coimbra, 3004-504 Coimbra, Portugal; Faculty of Pharmacy, University of Coimbra, 3000-548 Coimbra, Portugal., Rufino-Ramos D; Gene and Stem Cell Therapies for the Brain Group, Center for Neuroscience and Cell Biology, University of Coimbra (CNC-UC), 3004-504 Coimbra, Portugal; Center for Innovative Biomedicine and Biotechnology (CIBB), Vectors, Gene and Cell Therapy Group, University of Coimbra, 3004-504 Coimbra, Portugal; Faculty of Pharmacy, University of Coimbra, 3000-548 Coimbra, Portugal; Center for Genomic Medicine and Department of Pathology, Massachusetts General Hospital, Boston, MA 02115, USA; Department of Pathology, Massachusetts General Hospital, Boston, MA 02114, USA; Department of Pathology, Harvard Medical School, Boston, MA 02115, USA., Lopes MM; Gene and Stem Cell Therapies for the Brain Group, Center for Neuroscience and Cell Biology, University of Coimbra (CNC-UC), 3004-504 Coimbra, Portugal; Center for Innovative Biomedicine and Biotechnology (CIBB), Vectors, Gene and Cell Therapy Group, University of Coimbra, 3004-504 Coimbra, Portugal; GeneT, Center for Excellence in Gene Therapy in Portugal, University of Coimbra, 3004-504 Coimbra, Portugal; ViraVector-Viral Vectors for Gene Transfer Core Facility, University of Coimbra, 3004-504 Coimbra, Portugal; Institute for Interdisciplinary Research (iiiUC), University of Coimbra, 3030-789 Coimbra, Portugal., Leandro K; Gene and Stem Cell Therapies for the Brain Group, Center for Neuroscience and Cell Biology, University of Coimbra (CNC-UC), 3004-504 Coimbra, Portugal; Center for Innovative Biomedicine and Biotechnology (CIBB), Vectors, Gene and Cell Therapy Group, University of Coimbra, 3004-504 Coimbra, Portugal; GeneT, Center for Excellence in Gene Therapy in Portugal, University of Coimbra, 3004-504 Coimbra, Portugal; Faculty of Pharmacy, University of Coimbra, 3000-548 Coimbra, Portugal., Miranda CO; Gene and Stem Cell Therapies for the Brain Group, Center for Neuroscience and Cell Biology, University of Coimbra (CNC-UC), 3004-504 Coimbra, Portugal; Center for Innovative Biomedicine and Biotechnology (CIBB), Vectors, Gene and Cell Therapy Group, University of Coimbra, 3004-504 Coimbra, Portugal; GeneT, Center for Excellence in Gene Therapy in Portugal, University of Coimbra, 3004-504 Coimbra, Portugal; Institute for Interdisciplinary Research (iiiUC), University of Coimbra, 3030-789 Coimbra, Portugal., Almeida R; Gene and Stem Cell Therapies for the Brain Group, Center for Neuroscience and Cell Biology, University of Coimbra (CNC-UC), 3004-504 Coimbra, Portugal; Center for Innovative Biomedicine and Biotechnology (CIBB), Vectors, Gene and Cell Therapy Group, University of Coimbra, 3004-504 Coimbra, Portugal; GeneT, Center for Excellence in Gene Therapy in Portugal, University of Coimbra, 3004-504 Coimbra, Portugal; ViraVector-Viral Vectors for Gene Transfer Core Facility, University of Coimbra, 3004-504 Coimbra, Portugal., Lopes-Gabriel G; Gene and Stem Cell Therapies for the Brain Group, Center for Neuroscience and Cell Biology, University of Coimbra (CNC-UC), 3004-504 Coimbra, Portugal; Center for Innovative Biomedicine and Biotechnology (CIBB), Vectors, Gene and Cell Therapy Group, University of Coimbra, 3004-504 Coimbra, Portugal; GeneT, Center for Excellence in Gene Therapy in Portugal, University of Coimbra, 3004-504 Coimbra, Portugal; ViraVector-Viral Vectors for Gene Transfer Core Facility, University of Coimbra, 3004-504 Coimbra, Portugal; Faculty of Pharmacy, University of Coimbra, 3000-548 Coimbra, Portugal., de Sousa-Lourenço J; Gene and Stem Cell Therapies for the Brain Group, Center for Neuroscience and Cell Biology, University of Coimbra (CNC-UC), 3004-504 Coimbra, Portugal; Center for Innovative Biomedicine and Biotechnology (CIBB), Vectors, Gene and Cell Therapy Group, University of Coimbra, 3004-504 Coimbra, Portugal; GeneT, Center for Excellence in Gene Therapy in Portugal, University of Coimbra, 3004-504 Coimbra, Portugal; ViraVector-Viral Vectors for Gene Transfer Core Facility, University of Coimbra, 3004-504 Coimbra, Portugal., Lopes SM; Gene and Stem Cell Therapies for the Brain Group, Center for Neuroscience and Cell Biology, University of Coimbra (CNC-UC), 3004-504 Coimbra, Portugal; Center for Innovative Biomedicine and Biotechnology (CIBB), Vectors, Gene and Cell Therapy Group, University of Coimbra, 3004-504 Coimbra, Portugal; GeneT, Center for Excellence in Gene Therapy in Portugal, University of Coimbra, 3004-504 Coimbra, Portugal; Institute for Interdisciplinary Research (iiiUC), University of Coimbra, 3030-789 Coimbra, Portugal., Gaspar LS; Gene and Stem Cell Therapies for the Brain Group, Center for Neuroscience and Cell Biology, University of Coimbra (CNC-UC), 3004-504 Coimbra, Portugal; Center for Innovative Biomedicine and Biotechnology (CIBB), Vectors, Gene and Cell Therapy Group, University of Coimbra, 3004-504 Coimbra, Portugal; GeneT, Center for Excellence in Gene Therapy in Portugal, University of Coimbra, 3004-504 Coimbra, Portugal; Institute for Interdisciplinary Research (iiiUC), University of Coimbra, 3030-789 Coimbra, Portugal., Lobo DD; Gene and Stem Cell Therapies for the Brain Group, Center for Neuroscience and Cell Biology, University of Coimbra (CNC-UC), 3004-504 Coimbra, Portugal; Center for Innovative Biomedicine and Biotechnology (CIBB), Vectors, Gene and Cell Therapy Group, University of Coimbra, 3004-504 Coimbra, Portugal; GeneT, Center for Excellence in Gene Therapy in Portugal, University of Coimbra, 3004-504 Coimbra, Portugal; Institute for Interdisciplinary Research (iiiUC), University of Coimbra, 3030-789 Coimbra, Portugal., Silva AC; Gene and Stem Cell Therapies for the Brain Group, Center for Neuroscience and Cell Biology, University of Coimbra (CNC-UC), 3004-504 Coimbra, Portugal; Center for Innovative Biomedicine and Biotechnology (CIBB), Vectors, Gene and Cell Therapy Group, University of Coimbra, 3004-504 Coimbra, Portugal; GeneT, Center for Excellence in Gene Therapy in Portugal, University of Coimbra, 3004-504 Coimbra, Portugal; Institute for Interdisciplinary Research (iiiUC), University of Coimbra, 3030-789 Coimbra, Portugal., Ribeiro-Rodrigues TM; Center for Innovative Biomedicine and Biotechnology (CIBB), Vectors, Gene and Cell Therapy Group, University of Coimbra, 3004-504 Coimbra, Portugal; Coimbra Institute for Clinical and Biomedical Research (iCBR), University of Coimbra, 3004-504 Coimbra, Portugal; Clinical Academic Center of Coimbra (CACC), Coimbra, Portugal; Faculty of Medicine, University of Coimbra, 3000-548 Coimbra, Portugal., Girão H; Center for Innovative Biomedicine and Biotechnology (CIBB), Vectors, Gene and Cell Therapy Group, University of Coimbra, 3004-504 Coimbra, Portugal; Coimbra Institute for Clinical and Biomedical Research (iCBR), University of Coimbra, 3004-504 Coimbra, Portugal; Clinical Academic Center of Coimbra (CACC), Coimbra, Portugal; Faculty of Medicine, University of Coimbra, 3000-548 Coimbra, Portugal., Gomes CM; Center for Innovative Biomedicine and Biotechnology (CIBB), Vectors, Gene and Cell Therapy Group, University of Coimbra, 3004-504 Coimbra, Portugal; Coimbra Institute for Clinical and Biomedical Research (iCBR), University of Coimbra, 3004-504 Coimbra, Portugal; Clinical Academic Center of Coimbra (CACC), Coimbra, Portugal; Faculty of Medicine, University of Coimbra, 3000-548 Coimbra, Portugal., Baganha R; Gene and Stem Cell Therapies for the Brain Group, Center for Neuroscience and Cell Biology, University of Coimbra (CNC-UC), 3004-504 Coimbra, Portugal; Center for Innovative Biomedicine and Biotechnology (CIBB), Vectors, Gene and Cell Therapy Group, University of Coimbra, 3004-504 Coimbra, Portugal; GeneT, Center for Excellence in Gene Therapy in Portugal, University of Coimbra, 3004-504 Coimbra, Portugal; ViraVector-Viral Vectors for Gene Transfer Core Facility, University of Coimbra, 3004-504 Coimbra, Portugal; Institute for Interdisciplinary Research (iiiUC), University of Coimbra, 3030-789 Coimbra, Portugal., Duarte S; Gene and Stem Cell Therapies for the Brain Group, Center for Neuroscience and Cell Biology, University of Coimbra (CNC-UC), 3004-504 Coimbra, Portugal; Center for Innovative Biomedicine and Biotechnology (CIBB), Vectors, Gene and Cell Therapy Group, University of Coimbra, 3004-504 Coimbra, Portugal; GeneT, Center for Excellence in Gene Therapy in Portugal, University of Coimbra, 3004-504 Coimbra, Portugal; Institute for Interdisciplinary Research (iiiUC), University of Coimbra, 3030-789 Coimbra, Portugal., Maguire CA; Molecular Neurogenetics Unit, Massachusetts General Hospital, Harvard Medical School, Charlestown, MA 02129, USA., Santana MM; Gene and Stem Cell Therapies for the Brain Group, Center for Neuroscience and Cell Biology, University of Coimbra (CNC-UC), 3004-504 Coimbra, Portugal; Center for Innovative Biomedicine and Biotechnology (CIBB), Vectors, Gene and Cell Therapy Group, University of Coimbra, 3004-504 Coimbra, Portugal; GeneT, Center for Excellence in Gene Therapy in Portugal, University of Coimbra, 3004-504 Coimbra, Portugal; Institute for Interdisciplinary Research (iiiUC), University of Coimbra, 3030-789 Coimbra, Portugal., Pereira de Almeida L; Gene and Stem Cell Therapies for the Brain Group, Center for Neuroscience and Cell Biology, University of Coimbra (CNC-UC), 3004-504 Coimbra, Portugal; Center for Innovative Biomedicine and Biotechnology (CIBB), Vectors, Gene and Cell Therapy Group, University of Coimbra, 3004-504 Coimbra, Portugal; GeneT, Center for Excellence in Gene Therapy in Portugal, University of Coimbra, 3004-504 Coimbra, Portugal; ViraVector-Viral Vectors for Gene Transfer Core Facility, University of Coimbra, 3004-504 Coimbra, Portugal; Faculty of Pharmacy, University of Coimbra, 3000-548 Coimbra, Portugal. Electronic address: luispa@cnc.uc.pt., Nobre RJ; Gene and Stem Cell Therapies for the Brain Group, Center for Neuroscience and Cell Biology, University of Coimbra (CNC-UC), 3004-504 Coimbra, Portugal; Center for Innovative Biomedicine and Biotechnology (CIBB), Vectors, Gene and Cell Therapy Group, University of Coimbra, 3004-504 Coimbra, Portugal; GeneT, Center for Excellence in Gene Therapy in Portugal, University of Coimbra, 3004-504 Coimbra, Portugal; ViraVector-Viral Vectors for Gene Transfer Core Facility, University of Coimbra, 3004-504 Coimbra, Portugal; Institute for Interdisciplinary Research (iiiUC), University of Coimbra, 3030-789 Coimbra, Portugal. Electronic address: rui.nobre@cnc.uc.pt.
Source: Molecular therapy : the journal of the American Society of Gene Therapy [Mol Ther] 2026 Jan 07; Vol. 34 (1), pp. 161-178. Date of Electronic Publication: 2025 Oct 11.
Publication Type: Journal Article
Journal Info: Publisher: Cell Press Country of Publication: United States NLM ID: 100890581 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1525-0024 (Electronic) Linking ISSN: 15250016 NLM ISO Abbreviation: Mol Ther Subsets: MEDLINE
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  Data: Extracellular vesicles-associated AAVs for the treatment of Machado-Joseph disease.
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  Data: <searchLink fieldCode="AU" term="%22Henriques+C%22">Henriques C</searchLink>; Gene and Stem Cell Therapies for the Brain Group, Center for Neuroscience and Cell Biology, University of Coimbra (CNC-UC), 3004-504 Coimbra, Portugal; Center for Innovative Biomedicine and Biotechnology (CIBB), Vectors, Gene and Cell Therapy Group, University of Coimbra, 3004-504 Coimbra, Portugal; GeneT, Center for Excellence in Gene Therapy in Portugal, University of Coimbra, 3004-504 Coimbra, Portugal; ViraVector-Viral Vectors for Gene Transfer Core Facility, University of Coimbra, 3004-504 Coimbra, Portugal; Faculty of Pharmacy, University of Coimbra, 3000-548 Coimbra, Portugal.<br /><searchLink fieldCode="AU" term="%22Albuquerque+P%22">Albuquerque P</searchLink>; Gene and Stem Cell Therapies for the Brain Group, Center for Neuroscience and Cell Biology, University of Coimbra (CNC-UC), 3004-504 Coimbra, Portugal; Center for Innovative Biomedicine and Biotechnology (CIBB), Vectors, Gene and Cell Therapy Group, University of Coimbra, 3004-504 Coimbra, Portugal; Faculty of Pharmacy, University of Coimbra, 3000-548 Coimbra, Portugal.<br /><searchLink fieldCode="AU" term="%22Rufino-Ramos+D%22">Rufino-Ramos D</searchLink>; Gene and Stem Cell Therapies for the Brain Group, Center for Neuroscience and Cell Biology, University of Coimbra (CNC-UC), 3004-504 Coimbra, Portugal; Center for Innovative Biomedicine and Biotechnology (CIBB), Vectors, Gene and Cell Therapy Group, University of Coimbra, 3004-504 Coimbra, Portugal; Faculty of Pharmacy, University of Coimbra, 3000-548 Coimbra, Portugal; Center for Genomic Medicine and Department of Pathology, Massachusetts General Hospital, Boston, MA 02115, USA; Department of Pathology, Massachusetts General Hospital, Boston, MA 02114, USA; Department of Pathology, Harvard Medical School, Boston, MA 02115, USA.<br /><searchLink fieldCode="AU" term="%22Lopes+MM%22">Lopes MM</searchLink>; Gene and Stem Cell Therapies for the Brain Group, Center for Neuroscience and Cell Biology, University of Coimbra (CNC-UC), 3004-504 Coimbra, Portugal; Center for Innovative Biomedicine and Biotechnology (CIBB), Vectors, Gene and Cell Therapy Group, University of Coimbra, 3004-504 Coimbra, Portugal; GeneT, Center for Excellence in Gene Therapy in Portugal, University of Coimbra, 3004-504 Coimbra, Portugal; ViraVector-Viral Vectors for Gene Transfer Core Facility, University of Coimbra, 3004-504 Coimbra, Portugal; Institute for Interdisciplinary Research (iiiUC), University of Coimbra, 3030-789 Coimbra, Portugal.<br /><searchLink fieldCode="AU" term="%22Leandro+K%22">Leandro K</searchLink>; Gene and Stem Cell Therapies for the Brain Group, Center for Neuroscience and Cell Biology, University of Coimbra (CNC-UC), 3004-504 Coimbra, Portugal; Center for Innovative Biomedicine and Biotechnology (CIBB), Vectors, Gene and Cell Therapy Group, University of Coimbra, 3004-504 Coimbra, Portugal; GeneT, Center for Excellence in Gene Therapy in Portugal, University of Coimbra, 3004-504 Coimbra, Portugal; Faculty of Pharmacy, University of Coimbra, 3000-548 Coimbra, Portugal.<br /><searchLink fieldCode="AU" term="%22Miranda+CO%22">Miranda CO</searchLink>; Gene and Stem Cell Therapies for the Brain Group, Center for Neuroscience and Cell Biology, University of Coimbra (CNC-UC), 3004-504 Coimbra, Portugal; Center for Innovative Biomedicine and Biotechnology (CIBB), Vectors, Gene and Cell Therapy Group, University of Coimbra, 3004-504 Coimbra, Portugal; GeneT, Center for Excellence in Gene Therapy in Portugal, University of Coimbra, 3004-504 Coimbra, Portugal; Institute for Interdisciplinary Research (iiiUC), University of Coimbra, 3030-789 Coimbra, Portugal.<br /><searchLink fieldCode="AU" term="%22Almeida+R%22">Almeida R</searchLink>; Gene and Stem Cell Therapies for the Brain Group, Center for Neuroscience and Cell Biology, University of Coimbra (CNC-UC), 3004-504 Coimbra, Portugal; Center for Innovative Biomedicine and Biotechnology (CIBB), Vectors, Gene and Cell Therapy Group, University of Coimbra, 3004-504 Coimbra, Portugal; GeneT, Center for Excellence in Gene Therapy in Portugal, University of Coimbra, 3004-504 Coimbra, Portugal; ViraVector-Viral Vectors for Gene Transfer Core Facility, University of Coimbra, 3004-504 Coimbra, Portugal.<br /><searchLink fieldCode="AU" term="%22Lopes-Gabriel+G%22">Lopes-Gabriel G</searchLink>; Gene and Stem Cell Therapies for the Brain Group, Center for Neuroscience and Cell Biology, University of Coimbra (CNC-UC), 3004-504 Coimbra, Portugal; Center for Innovative Biomedicine and Biotechnology (CIBB), Vectors, Gene and Cell Therapy Group, University of Coimbra, 3004-504 Coimbra, Portugal; GeneT, Center for Excellence in Gene Therapy in Portugal, University of Coimbra, 3004-504 Coimbra, Portugal; ViraVector-Viral Vectors for Gene Transfer Core Facility, University of Coimbra, 3004-504 Coimbra, Portugal; Faculty of Pharmacy, University of Coimbra, 3000-548 Coimbra, Portugal.<br /><searchLink fieldCode="AU" term="%22de+Sousa-Lourenço+J%22">de Sousa-Lourenço J</searchLink>; Gene and Stem Cell Therapies for the Brain Group, Center for Neuroscience and Cell Biology, University of Coimbra (CNC-UC), 3004-504 Coimbra, Portugal; Center for Innovative Biomedicine and Biotechnology (CIBB), Vectors, Gene and Cell Therapy Group, University of Coimbra, 3004-504 Coimbra, Portugal; GeneT, Center for Excellence in Gene Therapy in Portugal, University of Coimbra, 3004-504 Coimbra, Portugal; ViraVector-Viral Vectors for Gene Transfer Core Facility, University of Coimbra, 3004-504 Coimbra, Portugal.<br /><searchLink fieldCode="AU" term="%22Lopes+SM%22">Lopes SM</searchLink>; Gene and Stem Cell Therapies for the Brain Group, Center for Neuroscience and Cell Biology, University of Coimbra (CNC-UC), 3004-504 Coimbra, Portugal; Center for Innovative Biomedicine and Biotechnology (CIBB), Vectors, Gene and Cell Therapy Group, University of Coimbra, 3004-504 Coimbra, Portugal; GeneT, Center for Excellence in Gene Therapy in Portugal, University of Coimbra, 3004-504 Coimbra, Portugal; Institute for Interdisciplinary Research (iiiUC), University of Coimbra, 3030-789 Coimbra, Portugal.<br /><searchLink fieldCode="AU" term="%22Gaspar+LS%22">Gaspar LS</searchLink>; Gene and Stem Cell Therapies for the Brain Group, Center for Neuroscience and Cell Biology, University of Coimbra (CNC-UC), 3004-504 Coimbra, Portugal; Center for Innovative Biomedicine and Biotechnology (CIBB), Vectors, Gene and Cell Therapy Group, University of Coimbra, 3004-504 Coimbra, Portugal; GeneT, Center for Excellence in Gene Therapy in Portugal, University of Coimbra, 3004-504 Coimbra, Portugal; Institute for Interdisciplinary Research (iiiUC), University of Coimbra, 3030-789 Coimbra, Portugal.<br /><searchLink fieldCode="AU" term="%22Lobo+DD%22">Lobo DD</searchLink>; Gene and Stem Cell Therapies for the Brain Group, Center for Neuroscience and Cell Biology, University of Coimbra (CNC-UC), 3004-504 Coimbra, Portugal; Center for Innovative Biomedicine and Biotechnology (CIBB), Vectors, Gene and Cell Therapy Group, University of Coimbra, 3004-504 Coimbra, Portugal; GeneT, Center for Excellence in Gene Therapy in Portugal, University of Coimbra, 3004-504 Coimbra, Portugal; Institute for Interdisciplinary Research (iiiUC), University of Coimbra, 3030-789 Coimbra, Portugal.<br /><searchLink fieldCode="AU" term="%22Silva+AC%22">Silva AC</searchLink>; Gene and Stem Cell Therapies for the Brain Group, Center for Neuroscience and Cell Biology, University of Coimbra (CNC-UC), 3004-504 Coimbra, Portugal; Center for Innovative Biomedicine and Biotechnology (CIBB), Vectors, Gene and Cell Therapy Group, University of Coimbra, 3004-504 Coimbra, Portugal; GeneT, Center for Excellence in Gene Therapy in Portugal, University of Coimbra, 3004-504 Coimbra, Portugal; Institute for Interdisciplinary Research (iiiUC), University of Coimbra, 3030-789 Coimbra, Portugal.<br /><searchLink fieldCode="AU" term="%22Ribeiro-Rodrigues+TM%22">Ribeiro-Rodrigues TM</searchLink>; Center for Innovative Biomedicine and Biotechnology (CIBB), Vectors, Gene and Cell Therapy Group, University of Coimbra, 3004-504 Coimbra, Portugal; Coimbra Institute for Clinical and Biomedical Research (iCBR), University of Coimbra, 3004-504 Coimbra, Portugal; Clinical Academic Center of Coimbra (CACC), Coimbra, Portugal; Faculty of Medicine, University of Coimbra, 3000-548 Coimbra, Portugal.<br /><searchLink fieldCode="AU" term="%22Girão+H%22">Girão H</searchLink>; Center for Innovative Biomedicine and Biotechnology (CIBB), Vectors, Gene and Cell Therapy Group, University of Coimbra, 3004-504 Coimbra, Portugal; Coimbra Institute for Clinical and Biomedical Research (iCBR), University of Coimbra, 3004-504 Coimbra, Portugal; Clinical Academic Center of Coimbra (CACC), Coimbra, Portugal; Faculty of Medicine, University of Coimbra, 3000-548 Coimbra, Portugal.<br /><searchLink fieldCode="AU" term="%22Gomes+CM%22">Gomes CM</searchLink>; Center for Innovative Biomedicine and Biotechnology (CIBB), Vectors, Gene and Cell Therapy Group, University of Coimbra, 3004-504 Coimbra, Portugal; Coimbra Institute for Clinical and Biomedical Research (iCBR), University of Coimbra, 3004-504 Coimbra, Portugal; Clinical Academic Center of Coimbra (CACC), Coimbra, Portugal; Faculty of Medicine, University of Coimbra, 3000-548 Coimbra, Portugal.<br /><searchLink fieldCode="AU" term="%22Baganha+R%22">Baganha R</searchLink>; Gene and Stem Cell Therapies for the Brain Group, Center for Neuroscience and Cell Biology, University of Coimbra (CNC-UC), 3004-504 Coimbra, Portugal; Center for Innovative Biomedicine and Biotechnology (CIBB), Vectors, Gene and Cell Therapy Group, University of Coimbra, 3004-504 Coimbra, Portugal; GeneT, Center for Excellence in Gene Therapy in Portugal, University of Coimbra, 3004-504 Coimbra, Portugal; ViraVector-Viral Vectors for Gene Transfer Core Facility, University of Coimbra, 3004-504 Coimbra, Portugal; Institute for Interdisciplinary Research (iiiUC), University of Coimbra, 3030-789 Coimbra, Portugal.<br /><searchLink fieldCode="AU" term="%22Duarte+S%22">Duarte S</searchLink>; Gene and Stem Cell Therapies for the Brain Group, Center for Neuroscience and Cell Biology, University of Coimbra (CNC-UC), 3004-504 Coimbra, Portugal; Center for Innovative Biomedicine and Biotechnology (CIBB), Vectors, Gene and Cell Therapy Group, University of Coimbra, 3004-504 Coimbra, Portugal; GeneT, Center for Excellence in Gene Therapy in Portugal, University of Coimbra, 3004-504 Coimbra, Portugal; Institute for Interdisciplinary Research (iiiUC), University of Coimbra, 3030-789 Coimbra, Portugal.<br /><searchLink fieldCode="AU" term="%22Maguire+CA%22">Maguire CA</searchLink>; Molecular Neurogenetics Unit, Massachusetts General Hospital, Harvard Medical School, Charlestown, MA 02129, USA.<br /><searchLink fieldCode="AU" term="%22Santana+MM%22">Santana MM</searchLink>; Gene and Stem Cell Therapies for the Brain Group, Center for Neuroscience and Cell Biology, University of Coimbra (CNC-UC), 3004-504 Coimbra, Portugal; Center for Innovative Biomedicine and Biotechnology (CIBB), Vectors, Gene and Cell Therapy Group, University of Coimbra, 3004-504 Coimbra, Portugal; GeneT, Center for Excellence in Gene Therapy in Portugal, University of Coimbra, 3004-504 Coimbra, Portugal; Institute for Interdisciplinary Research (iiiUC), University of Coimbra, 3030-789 Coimbra, Portugal.<br /><searchLink fieldCode="AU" term="%22Pereira+de+Almeida+L%22">Pereira de Almeida L</searchLink>; Gene and Stem Cell Therapies for the Brain Group, Center for Neuroscience and Cell Biology, University of Coimbra (CNC-UC), 3004-504 Coimbra, Portugal; Center for Innovative Biomedicine and Biotechnology (CIBB), Vectors, Gene and Cell Therapy Group, University of Coimbra, 3004-504 Coimbra, Portugal; GeneT, Center for Excellence in Gene Therapy in Portugal, University of Coimbra, 3004-504 Coimbra, Portugal; ViraVector-Viral Vectors for Gene Transfer Core Facility, University of Coimbra, 3004-504 Coimbra, Portugal; Faculty of Pharmacy, University of Coimbra, 3000-548 Coimbra, Portugal. Electronic address: luispa@cnc.uc.pt.<br /><searchLink fieldCode="AU" term="%22Nobre+RJ%22">Nobre RJ</searchLink>; Gene and Stem Cell Therapies for the Brain Group, Center for Neuroscience and Cell Biology, University of Coimbra (CNC-UC), 3004-504 Coimbra, Portugal; Center for Innovative Biomedicine and Biotechnology (CIBB), Vectors, Gene and Cell Therapy Group, University of Coimbra, 3004-504 Coimbra, Portugal; GeneT, Center for Excellence in Gene Therapy in Portugal, University of Coimbra, 3004-504 Coimbra, Portugal; ViraVector-Viral Vectors for Gene Transfer Core Facility, University of Coimbra, 3004-504 Coimbra, Portugal; Institute for Interdisciplinary Research (iiiUC), University of Coimbra, 3030-789 Coimbra, Portugal. Electronic address: rui.nobre@cnc.uc.pt.
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  Data: <searchLink fieldCode="JN" term="%22100890581%22">Molecular therapy : the journal of the American Society of Gene Therapy</searchLink> [Mol Ther] 2026 Jan 07; Vol. 34 (1), pp. 161-178. <i>Date of Electronic Publication: </i>2025 Oct 11.
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      – TitleFull: Extracellular vesicles-associated AAVs for the treatment of Machado-Joseph disease.
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            NameFull: Ribeiro-Rodrigues TM
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            NameFull: Girão H
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            NameFull: Gomes CM
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            NameFull: Baganha R
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            NameFull: Duarte S
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            NameFull: Maguire CA
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            NameFull: Santana MM
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            NameFull: Pereira de Almeida L
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          Name:
            NameFull: Nobre RJ
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      – BibEntity:
          Dates:
            – D: 07
              M: 01
              Text: 2026 Jan 07
              Type: published
              Y: 2026
          Identifiers:
            – Type: issn-electronic
              Value: 1525-0024
          Numbering:
            – Type: volume
              Value: 34
            – Type: issue
              Value: 1
          Titles:
            – TitleFull: Molecular therapy : the journal of the American Society of Gene Therapy
              Type: main
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