Treatment of skeletal and non-skeletal alterations of Mucopolysaccharidosis type IVA by AAV-mediated gene therapy.
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| Title: | Treatment of skeletal and non-skeletal alterations of Mucopolysaccharidosis type IVA by AAV-mediated gene therapy. |
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| Authors: | Bertolin J; Center of Animal Biotechnology and Gene Therapy, Bellaterra, Spain.; Department of Biochemistry and Molecular Biology, Universitat Autònoma de Barcelona, Bellaterra, Spain., Sánchez V; Center of Animal Biotechnology and Gene Therapy, Bellaterra, Spain.; Department of Biochemistry and Molecular Biology, Universitat Autònoma de Barcelona, Bellaterra, Spain., Ribera A; Center of Animal Biotechnology and Gene Therapy, Bellaterra, Spain.; Department of Biochemistry and Molecular Biology, Universitat Autònoma de Barcelona, Bellaterra, Spain.; Centro de Investigación Biomédica en Red de Diabetes y Enfermedades Metabólicas Asociadas (CIBERDEM), Madrid, Spain., Jaén ML; Center of Animal Biotechnology and Gene Therapy, Bellaterra, Spain.; Department of Biochemistry and Molecular Biology, Universitat Autònoma de Barcelona, Bellaterra, Spain., Garcia M; Center of Animal Biotechnology and Gene Therapy, Bellaterra, Spain.; Department of Biochemistry and Molecular Biology, Universitat Autònoma de Barcelona, Bellaterra, Spain.; Centro de Investigación Biomédica en Red de Diabetes y Enfermedades Metabólicas Asociadas (CIBERDEM), Madrid, Spain., Pujol A; Center of Animal Biotechnology and Gene Therapy, Bellaterra, Spain.; Department of Biochemistry and Molecular Biology, Universitat Autònoma de Barcelona, Bellaterra, Spain., Sánchez X; Center of Animal Biotechnology and Gene Therapy, Bellaterra, Spain.; Department of Biochemistry and Molecular Biology, Universitat Autònoma de Barcelona, Bellaterra, Spain., Muñoz S; Center of Animal Biotechnology and Gene Therapy, Bellaterra, Spain.; Department of Biochemistry and Molecular Biology, Universitat Autònoma de Barcelona, Bellaterra, Spain.; Centro de Investigación Biomédica en Red de Diabetes y Enfermedades Metabólicas Asociadas (CIBERDEM), Madrid, Spain., Marcó S; Center of Animal Biotechnology and Gene Therapy, Bellaterra, Spain.; Department of Biochemistry and Molecular Biology, Universitat Autònoma de Barcelona, Bellaterra, Spain., Pérez J; Center of Animal Biotechnology and Gene Therapy, Bellaterra, Spain.; Department of Biochemistry and Molecular Biology, Universitat Autònoma de Barcelona, Bellaterra, Spain., Elias G; Center of Animal Biotechnology and Gene Therapy, Bellaterra, Spain.; Department of Biochemistry and Molecular Biology, Universitat Autònoma de Barcelona, Bellaterra, Spain., León X; Center of Animal Biotechnology and Gene Therapy, Bellaterra, Spain.; Department of Biochemistry and Molecular Biology, Universitat Autònoma de Barcelona, Bellaterra, Spain.; Centro de Investigación Biomédica en Red de Diabetes y Enfermedades Metabólicas Asociadas (CIBERDEM), Madrid, Spain., Roca C; Center of Animal Biotechnology and Gene Therapy, Bellaterra, Spain.; Department of Biochemistry and Molecular Biology, Universitat Autònoma de Barcelona, Bellaterra, Spain.; Centro de Investigación Biomédica en Red de Diabetes y Enfermedades Metabólicas Asociadas (CIBERDEM), Madrid, Spain., Jimenez V; Center of Animal Biotechnology and Gene Therapy, Bellaterra, Spain.; Department of Biochemistry and Molecular Biology, Universitat Autònoma de Barcelona, Bellaterra, Spain.; Centro de Investigación Biomédica en Red de Diabetes y Enfermedades Metabólicas Asociadas (CIBERDEM), Madrid, Spain., Otaegui P; Center of Animal Biotechnology and Gene Therapy, Bellaterra, Spain., Mulero F; Molecular Imaging Unit, Spanish National Cancer Research Center (CNIO), Madrid, Spain., Navarro M; Center of Animal Biotechnology and Gene Therapy, Bellaterra, Spain.; Department of Animal Health and Anatomy, School of Veterinary Medicine, Universitat Autònoma de Barcelona, Bellaterra, Spain., Ruberte J; Center of Animal Biotechnology and Gene Therapy, Bellaterra, Spain.; Department of Animal Health and Anatomy, School of Veterinary Medicine, Universitat Autònoma de Barcelona, Bellaterra, Spain., Bosch F; Center of Animal Biotechnology and Gene Therapy, Bellaterra, Spain. fatima.bosch@uab.es.; Department of Biochemistry and Molecular Biology, Universitat Autònoma de Barcelona, Bellaterra, Spain. fatima.bosch@uab.es.; Centro de Investigación Biomédica en Red de Diabetes y Enfermedades Metabólicas Asociadas (CIBERDEM), Madrid, Spain. fatima.bosch@uab.es. |
| Source: | Nature communications [Nat Commun] 2021 Sep 09; Vol. 12 (1), pp. 5343. Date of Electronic Publication: 2021 Sep 09. |
| Publication Type: | Journal Article; Research Support, Non-U.S. Gov't |
| Journal Info: | Publisher: Nature Pub. Group Country of Publication: England NLM ID: 101528555 Publication Model: Electronic Cited Medium: Internet ISSN: 2041-1723 (Electronic) Linking ISSN: 20411723 NLM ISO Abbreviation: Nat Commun Subsets: MEDLINE |
| Database: | MEDLINE Ultimate |
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| ISSN: | 2041-1723 |
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| DOI: | 10.1038/s41467-021-25697-y |
