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Young mice administered adult doses of AAV5-hFVIII-SQ achieve therapeutic factor VIII expression into adulthood.
Zhang, Lening; Yates, Bridget; Murphy, Ryan; Liu, Su; Xie, Lin; Handyside, Britta; Sihn, Choong-Ryoul; Bouwman, Taren; Galicia, Nicole; Tan, Danielle; Fonck, Carlos; Arens, Jeremy; Clark, Annie; Zhang, Weiming; Chandra, Sundeep; Srimani, Jaydeep; Holcomb, Jennifer; Van Tuyl, Andrea; Henshaw, Joshua; Vettermann, Christian; Siso, Silvia; Su, Cheng; Bullens, Sherry; Bunting, Stuart; O'Neill, Charles; Fong, Sylvia.
Afiliación
  • Zhang L; Biology Research, BioMarin Pharmaceutical Inc., Novato, CA 94949, USA.
  • Yates B; Biology Research, BioMarin Pharmaceutical Inc., Novato, CA 94949, USA.
  • Murphy R; Biology Research, BioMarin Pharmaceutical Inc., Novato, CA 94949, USA.
  • Liu S; Biology Research, BioMarin Pharmaceutical Inc., Novato, CA 94949, USA.
  • Xie L; Biology Research, BioMarin Pharmaceutical Inc., Novato, CA 94949, USA.
  • Handyside B; Biology Research, BioMarin Pharmaceutical Inc., Novato, CA 94949, USA.
  • Sihn CR; Biology Research, BioMarin Pharmaceutical Inc., Novato, CA 94949, USA.
  • Bouwman T; Biology Research, BioMarin Pharmaceutical Inc., Novato, CA 94949, USA.
  • Galicia N; Biology Research, BioMarin Pharmaceutical Inc., Novato, CA 94949, USA.
  • Tan D; Biology Research, BioMarin Pharmaceutical Inc., Novato, CA 94949, USA.
  • Fonck C; Pharmacological Sciences, BioMarin Pharmaceutical Inc., Novato, CA 94949, USA.
  • Arens J; Translational Sciences, BioMarin Pharmaceutical Inc., Novato, CA 94949, USA.
  • Clark A; Translational Sciences, BioMarin Pharmaceutical Inc., Novato, CA 94949, USA.
  • Zhang W; Data Sciences and Analytics, BioMarin Pharmaceutical Inc., Novato, CA 94949, USA.
  • Chandra S; Translational Sciences, BioMarin Pharmaceutical Inc., Novato, CA 94949, USA.
  • Srimani J; Translational Sciences, BioMarin Pharmaceutical Inc., Novato, CA 94949, USA.
  • Holcomb J; Translational Sciences, BioMarin Pharmaceutical Inc., Novato, CA 94949, USA.
  • Van Tuyl A; Translational Sciences, BioMarin Pharmaceutical Inc., Novato, CA 94949, USA.
  • Henshaw J; Translational Sciences, BioMarin Pharmaceutical Inc., Novato, CA 94949, USA.
  • Vettermann C; Translational Sciences, BioMarin Pharmaceutical Inc., Novato, CA 94949, USA.
  • Siso S; Biology Research, BioMarin Pharmaceutical Inc., Novato, CA 94949, USA.
  • Su C; Data Sciences and Analytics, BioMarin Pharmaceutical Inc., Novato, CA 94949, USA.
  • Bullens S; Biology Research, BioMarin Pharmaceutical Inc., Novato, CA 94949, USA.
  • Bunting S; Biology Research, BioMarin Pharmaceutical Inc., Novato, CA 94949, USA.
  • O'Neill C; Translational Sciences, BioMarin Pharmaceutical Inc., Novato, CA 94949, USA.
  • Fong S; Biology Research, BioMarin Pharmaceutical Inc., Novato, CA 94949, USA.
Mol Ther Methods Clin Dev ; 26: 519-531, 2022 Sep 08.
Article en En | MEDLINE | ID: mdl-36092364
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Texto completo: 1 Colección: 01-internacional Banco de datos: MEDLINE Tipo de estudio: Prognostic_studies Idioma: En Revista: Mol Ther Methods Clin Dev Año: 2022 Tipo del documento: Article País de afiliación: Estados Unidos
Texto completo
Imprimir
XML
PubMed Links
Buscar en Google

Texto completo: 1 Colección: 01-internacional Banco de datos: MEDLINE Tipo de estudio: Prognostic_studies Idioma: En Revista: Mol Ther Methods Clin Dev Año: 2022 Tipo del documento: Article País de afiliación: Estados Unidos