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Disease correction by AAV-mediated gene therapy in a new mouse model of mucopolysaccharidosis type IIID.
Roca, Carles; Motas, Sandra; Marcó, Sara; Ribera, Albert; Sánchez, Víctor; Sánchez, Xavier; Bertolin, Joan; León, Xavier; Pérez, Jennifer; Garcia, Miguel; Villacampa, Pilar; Ruberte, Jesús; Pujol, Anna; Haurigot, Virginia; Bosch, Fatima.
Affiliation
  • Roca C; Center of Animal Biotechnology and Gene Therapy.
  • Motas S; Departments of Biochemistry and Molecular Biology.
  • Marcó S; Centro de Investigación Biomédica en Red de Diabetes y Enfermedades Metabólicas Asociadas (CIBERDEM), Barcelona 08036, Spain.
  • Ribera A; Center of Animal Biotechnology and Gene Therapy.
  • Sánchez V; Departments of Biochemistry and Molecular Biology.
  • Sánchez X; Center of Animal Biotechnology and Gene Therapy.
  • Bertolin J; Departments of Biochemistry and Molecular Biology.
  • León X; Center of Animal Biotechnology and Gene Therapy.
  • Pérez J; Departments of Biochemistry and Molecular Biology.
  • Garcia M; Centro de Investigación Biomédica en Red de Diabetes y Enfermedades Metabólicas Asociadas (CIBERDEM), Barcelona 08036, Spain.
  • Villacampa P; Center of Animal Biotechnology and Gene Therapy.
  • Ruberte J; Departments of Biochemistry and Molecular Biology.
  • Pujol A; Center of Animal Biotechnology and Gene Therapy.
  • Haurigot V; Departments of Biochemistry and Molecular Biology.
  • Bosch F; Center of Animal Biotechnology and Gene Therapy.
Hum Mol Genet ; 26(8): 1535-1551, 2017 04 15.
Article in En | MEDLINE | ID: mdl-28334745
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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Sulfatases / Genetic Therapy / Lysosomal Storage Diseases / Mucopolysaccharidosis III Limits: Animals / Humans Language: En Journal: Hum Mol Genet Journal subject: BIOLOGIA MOLECULAR / GENETICA MEDICA Year: 2017 Document type: Article Country of publication:
Fulltext
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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Sulfatases / Genetic Therapy / Lysosomal Storage Diseases / Mucopolysaccharidosis III Limits: Animals / Humans Language: En Journal: Hum Mol Genet Journal subject: BIOLOGIA MOLECULAR / GENETICA MEDICA Year: 2017 Document type: Article Country of publication: