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Normalization and improvement of CNS deficits in mice with Hurler syndrome after long-term peripheral delivery of BBB-targeted iduronidase.
El-Amouri, Salim S; Dai, Mei; Han, Jing-Fen; Brady, Roscoe O; Pan, Dao.
Afiliación
  • El-Amouri SS; Molecular and Cell Therapy Program, Division of Experimental Hematology and Cancer Biology, Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio, USA.
  • Dai M; Molecular and Cell Therapy Program, Division of Experimental Hematology and Cancer Biology, Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio, USA.
  • Han JF; Molecular and Cell Therapy Program, Division of Experimental Hematology and Cancer Biology, Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio, USA.
  • Brady RO; National Institute of Neurological Disorders and Stroke, National Institutes of Health, Bethesda, Maryland, USA.
  • Pan D; Molecular and Cell Therapy Program, Division of Experimental Hematology and Cancer Biology, Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio, USA; Department of Pediatrics, University of Cincinnati School of Medicine, Cincinnati, Ohio, USA. Electronic address: dao.pan@cchmc.org.
Mol Ther ; 22(12): 2028-2037, 2014 Dec.
Article en En | MEDLINE | ID: mdl-25088464
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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Encéfalo / Células Madre Hematopoyéticas / Mucopolisacaridosis I / Receptores de Péptidos / Iduronidasa Límite: Animals / Humans Idioma: En Revista: Mol Ther Asunto de la revista: BIOLOGIA MOLECULAR / TERAPEUTICA Año: 2014 Tipo del documento: Article País de afiliación: Estados Unidos
Texto completo
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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Encéfalo / Células Madre Hematopoyéticas / Mucopolisacaridosis I / Receptores de Péptidos / Iduronidasa Límite: Animals / Humans Idioma: En Revista: Mol Ther Asunto de la revista: BIOLOGIA MOLECULAR / TERAPEUTICA Año: 2014 Tipo del documento: Article País de afiliación: Estados Unidos