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Preclinical assessment of an optimized AAV-FVIII vector in mice and non-human primates for the treatment of hemophilia A.
Elkouby, Liron; Armour, Sean M; Toso, Raffaella; DiPietro, Marti; Davidson, Robert J; Nguyen, Giang N; Willet, Mallory; Kutza, Stephanie; Silverberg, Joseph; Frick, Jennifer; Crosariol, Marco; Wang, Yuhuan; Wang, Chuansong; High, Katherine A; Sabatino, Denise E; Anguela, Xavier M.
  • Elkouby L; The Raymond G. Perelman Center for Cellular and Molecular Therapeutics, The Children's Hospital of Philadelphia, Colket Translational Research Building, Rm 5020, Philadelphia, PA 19104, USA.
  • Armour SM; Spark Therapeutics, Inc., Philadelphia, PA, USA.
  • Toso R; Spark Therapeutics, Inc., Philadelphia, PA, USA.
  • DiPietro M; Spark Therapeutics, Inc., Philadelphia, PA, USA.
  • Davidson RJ; Spark Therapeutics, Inc., Philadelphia, PA, USA.
  • Nguyen GN; The Raymond G. Perelman Center for Cellular and Molecular Therapeutics, The Children's Hospital of Philadelphia, Colket Translational Research Building, Rm 5020, Philadelphia, PA 19104, USA.
  • Willet M; The Raymond G. Perelman Center for Cellular and Molecular Therapeutics, The Children's Hospital of Philadelphia, Colket Translational Research Building, Rm 5020, Philadelphia, PA 19104, USA.
  • Kutza S; Spark Therapeutics, Inc., Philadelphia, PA, USA.
  • Silverberg J; Spark Therapeutics, Inc., Philadelphia, PA, USA.
  • Frick J; Spark Therapeutics, Inc., Philadelphia, PA, USA.
  • Crosariol M; Spark Therapeutics, Inc., Philadelphia, PA, USA.
  • Wang Y; Spark Therapeutics, Inc., Philadelphia, PA, USA.
  • Wang C; Spark Therapeutics, Inc., Philadelphia, PA, USA.
  • High KA; Spark Therapeutics, Inc., Philadelphia, PA, USA.
  • Sabatino DE; Spark Therapeutics, Inc., Philadelphia, PA, USA.
  • Anguela XM; The Raymond G. Perelman Center for Cellular and Molecular Therapeutics, The Children's Hospital of Philadelphia, Colket Translational Research Building, Rm 5020, Philadelphia, PA 19104, USA.
Mol Ther Methods Clin Dev ; 24: 20-29, 2022 Mar 10.
Article en En | MEDLINE | ID: mdl-34977269
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Texto completo: 1 Banco de datos: MEDLINE Tipo de estudio: Prognostic_studies Idioma: En Año: 2022 Tipo del documento: Article
Texto completo
Imprimir
XML
PubMed Links
Search on Google

Texto completo: 1 Banco de datos: MEDLINE Tipo de estudio: Prognostic_studies Idioma: En Año: 2022 Tipo del documento: Article