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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.
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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  Data: Treatment of skeletal and non-skeletal alterations of Mucopolysaccharidosis type IVA by AAV-mediated gene therapy.
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  Data: <searchLink fieldCode="AU" term="%22Bertolin+J%22">Bertolin J</searchLink>; Center of Animal Biotechnology and Gene Therapy, Bellaterra, Spain.; Department of Biochemistry and Molecular Biology, Universitat Autònoma de Barcelona, Bellaterra, Spain.<br /><searchLink fieldCode="AU" term="%22Sánchez+V%22">Sánchez V</searchLink>; Center of Animal Biotechnology and Gene Therapy, Bellaterra, Spain.; Department of Biochemistry and Molecular Biology, Universitat Autònoma de Barcelona, Bellaterra, Spain.<br /><searchLink fieldCode="AU" term="%22Ribera+A%22">Ribera A</searchLink>; 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.<br /><searchLink fieldCode="AU" term="%22Jaén+ML%22">Jaén ML</searchLink>; Center of Animal Biotechnology and Gene Therapy, Bellaterra, Spain.; Department of Biochemistry and Molecular Biology, Universitat Autònoma de Barcelona, Bellaterra, Spain.<br /><searchLink fieldCode="AU" term="%22Garcia+M%22">Garcia M</searchLink>; 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.<br /><searchLink fieldCode="AU" term="%22Pujol+A%22">Pujol A</searchLink>; Center of Animal Biotechnology and Gene Therapy, Bellaterra, Spain.; Department of Biochemistry and Molecular Biology, Universitat Autònoma de Barcelona, Bellaterra, Spain.<br /><searchLink fieldCode="AU" term="%22Sánchez+X%22">Sánchez X</searchLink>; Center of Animal Biotechnology and Gene Therapy, Bellaterra, Spain.; Department of Biochemistry and Molecular Biology, Universitat Autònoma de Barcelona, Bellaterra, Spain.<br /><searchLink fieldCode="AU" term="%22Muñoz+S%22">Muñoz S</searchLink>; 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.<br /><searchLink fieldCode="AU" term="%22Marcó+S%22">Marcó S</searchLink>; Center of Animal Biotechnology and Gene Therapy, Bellaterra, Spain.; Department of Biochemistry and Molecular Biology, Universitat Autònoma de Barcelona, Bellaterra, Spain.<br /><searchLink fieldCode="AU" term="%22Pérez+J%22">Pérez J</searchLink>; Center of Animal Biotechnology and Gene Therapy, Bellaterra, Spain.; Department of Biochemistry and Molecular Biology, Universitat Autònoma de Barcelona, Bellaterra, Spain.<br /><searchLink fieldCode="AU" term="%22Elias+G%22">Elias G</searchLink>; Center of Animal Biotechnology and Gene Therapy, Bellaterra, Spain.; Department of Biochemistry and Molecular Biology, Universitat Autònoma de Barcelona, Bellaterra, Spain.<br /><searchLink fieldCode="AU" term="%22León+X%22">León X</searchLink>; 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.<br /><searchLink fieldCode="AU" term="%22Roca+C%22">Roca C</searchLink>; 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.<br /><searchLink fieldCode="AU" term="%22Jimenez+V%22">Jimenez V</searchLink>; 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.<br /><searchLink fieldCode="AU" term="%22Otaegui+P%22">Otaegui P</searchLink>; Center of Animal Biotechnology and Gene Therapy, Bellaterra, Spain.<br /><searchLink fieldCode="AU" term="%22Mulero+F%22">Mulero F</searchLink>; Molecular Imaging Unit, Spanish National Cancer Research Center (CNIO), Madrid, Spain.<br /><searchLink fieldCode="AU" term="%22Navarro+M%22">Navarro M</searchLink>; 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.<br /><searchLink fieldCode="AU" term="%22Ruberte+J%22">Ruberte J</searchLink>; 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.<br /><searchLink fieldCode="AU" term="%22Bosch+F%22">Bosch F</searchLink>; 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.
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  Data: <searchLink fieldCode="JN" term="%22101528555%22">Nature communications</searchLink> [Nat Commun] 2021 Sep 09; Vol. 12 (1), pp. 5343. <i>Date of Electronic Publication: </i>2021 Sep 09.
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