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Seven-year follow-up of durability and safety of AAV CNS gene therapy for a lysosomal storage disorder in a large animal.
Marcó, Sara; Haurigot, Virginia; Jaén, Maria Luisa; Ribera, Albert; Sánchez, Víctor; Molas, Maria; Garcia, Miguel; León, Xavier; Roca, Carles; Sánchez, Xavier; Bertolin, Joan; Pérez, Jennifer; Elias, Gemma; Navarro, Marc; Carretero, Ana; Pumarola, Martí; Andaluz, Anna; Espada, Yvonne; Añor, Sonia; Bosch, Fatima.
Afiliação
  • Marcó S; Center of Animal Biotechnology and Gene Therapy, Edifici H, Universitat Autònoma de Barcelona, 08193 Bellaterra, Spain.
  • Haurigot V; Department of Biochemistry and Molecular Biology, Universitat Autònoma de Barcelona, 08193 Bellaterra, Spain.
  • Jaén ML; CIBER de Diabetes y Enfermedades Metabólicas Asociadas (CIBERDEM), 28029 Madrid, Spain.
  • Ribera A; Center of Animal Biotechnology and Gene Therapy, Edifici H, Universitat Autònoma de Barcelona, 08193 Bellaterra, Spain.
  • Sánchez V; Department of Biochemistry and Molecular Biology, Universitat Autònoma de Barcelona, 08193 Bellaterra, Spain.
  • Molas M; CIBER de Diabetes y Enfermedades Metabólicas Asociadas (CIBERDEM), 28029 Madrid, Spain.
  • Garcia M; Center of Animal Biotechnology and Gene Therapy, Edifici H, Universitat Autònoma de Barcelona, 08193 Bellaterra, Spain.
  • León X; Department of Biochemistry and Molecular Biology, Universitat Autònoma de Barcelona, 08193 Bellaterra, Spain.
  • Roca C; CIBER de Diabetes y Enfermedades Metabólicas Asociadas (CIBERDEM), 28029 Madrid, Spain.
  • Sánchez X; Center of Animal Biotechnology and Gene Therapy, Edifici H, Universitat Autònoma de Barcelona, 08193 Bellaterra, Spain.
  • Bertolin J; Department of Biochemistry and Molecular Biology, Universitat Autònoma de Barcelona, 08193 Bellaterra, Spain.
  • Pérez J; CIBER de Diabetes y Enfermedades Metabólicas Asociadas (CIBERDEM), 28029 Madrid, Spain.
  • Elias G; Center of Animal Biotechnology and Gene Therapy, Edifici H, Universitat Autònoma de Barcelona, 08193 Bellaterra, Spain.
  • Navarro M; Department of Biochemistry and Molecular Biology, Universitat Autònoma de Barcelona, 08193 Bellaterra, Spain.
  • Carretero A; Center of Animal Biotechnology and Gene Therapy, Edifici H, Universitat Autònoma de Barcelona, 08193 Bellaterra, Spain.
  • Pumarola M; Department of Biochemistry and Molecular Biology, Universitat Autònoma de Barcelona, 08193 Bellaterra, Spain.
  • Andaluz A; CIBER de Diabetes y Enfermedades Metabólicas Asociadas (CIBERDEM), 28029 Madrid, Spain.
  • Espada Y; Center of Animal Biotechnology and Gene Therapy, Edifici H, Universitat Autònoma de Barcelona, 08193 Bellaterra, Spain.
  • Añor S; Department of Biochemistry and Molecular Biology, Universitat Autònoma de Barcelona, 08193 Bellaterra, Spain.
  • Bosch F; CIBER de Diabetes y Enfermedades Metabólicas Asociadas (CIBERDEM), 28029 Madrid, Spain.
Mol Ther Methods Clin Dev ; 23: 370-389, 2021 Dec 10.
Article em En | MEDLINE | ID: mdl-34761052
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Texto completo: 1 Base de dados: MEDLINE Idioma: En Ano de publicação: 2021 Tipo de documento: Article
Texto completo
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XML
PubMed Links
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Texto completo: 1 Base de dados: MEDLINE Idioma: En Ano de publicação: 2021 Tipo de documento: Article