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Cerebrospinal Fluid-Directed rAAV9-rsATP7A Plus Subcutaneous Copper Histidinate Advance Survival and Outcomes in a Menkes Disease Mouse Model.
Haddad, Marie Reine; Choi, Eun-Young; Zerfas, Patricia M; Yi, Ling; Martinelli, Diego; Sullivan, Patricia; Goldstein, David S; Centeno, Jose A; Brinster, Lauren R; Ralle, Martina; Kaler, Stephen G.
Afiliação
  • Haddad MR; Section on Translational Neuroscience, Molecular Medicine Branch, Eunice Kennedy Shriver National Institute of Child Health and Human Development, Bethesda, MD, USA.
  • Choi EY; Section on Translational Neuroscience, Molecular Medicine Branch, Eunice Kennedy Shriver National Institute of Child Health and Human Development, Bethesda, MD, USA.
  • Zerfas PM; Diagnostic and Research Services Branch, Office of Research Services, Bethesda, MD, USA.
  • Yi L; Section on Translational Neuroscience, Molecular Medicine Branch, Eunice Kennedy Shriver National Institute of Child Health and Human Development, Bethesda, MD, USA.
  • Martinelli D; Section on Translational Neuroscience, Molecular Medicine Branch, Eunice Kennedy Shriver National Institute of Child Health and Human Development, Bethesda, MD, USA.
  • Sullivan P; Clinical Neurocardiology Section, National Institute of Neurological Disorders and Stroke, NIH, Bethesda, MD, USA.
  • Goldstein DS; Clinical Neurocardiology Section, National Institute of Neurological Disorders and Stroke, NIH, Bethesda, MD, USA.
  • Centeno JA; Division of Biology, Chemistry and Materials Science. Office of Science and Engineering Laboratories (OSEL), US Food and Drug Administration, Silver Spring, MD, USA.
  • Brinster LR; Diagnostic and Research Services Branch, Office of Research Services, Bethesda, MD, USA.
  • Ralle M; Department of Molecular and Medical Genetics, Oregon Health and Science University, Portland, OR 97239, USA.
  • Kaler SG; Section on Translational Neuroscience, Molecular Medicine Branch, Eunice Kennedy Shriver National Institute of Child Health and Human Development, Bethesda, MD, USA.
Mol Ther Methods Clin Dev ; 10: 165-178, 2018 Sep 21.
Article em En | MEDLINE | ID: mdl-30090842

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Tipo de estudo: Prognostic_studies Idioma: En Revista: Mol Ther Methods Clin Dev Ano de publicação: 2018 Tipo de documento: Article País de afiliação: Estados Unidos País de publicação: Estados Unidos

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Tipo de estudo: Prognostic_studies Idioma: En Revista: Mol Ther Methods Clin Dev Ano de publicação: 2018 Tipo de documento: Article País de afiliação: Estados Unidos País de publicação: Estados Unidos