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Efficacy and Safety of a Krabbe Disease Gene Therapy.
Hordeaux, Juliette; Jeffrey, Brianne A; Jian, Jinlong; Choudhury, Gourav R; Michalson, Kristofer; Mitchell, Thomas W; Buza, Elizabeth L; Chichester, Jessica; Dyer, Cecilia; Bagel, Jessica; Vite, Charles H; Bradbury, Allison M; Wilson, James M.
Afiliación
  • Hordeaux J; Gene Therapy Program, Department of Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, USA.
  • Jeffrey BA; Gene Therapy Program, Department of Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, USA.
  • Jian J; Gene Therapy Program, Department of Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, USA.
  • Choudhury GR; Gene Therapy Program, Department of Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, USA.
  • Michalson K; Gene Therapy Program, Department of Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, USA.
  • Mitchell TW; Gene Therapy Program, Department of Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, USA.
  • Buza EL; Gene Therapy Program, Department of Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, USA.
  • Chichester J; Gene Therapy Program, Department of Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, USA.
  • Dyer C; Gene Therapy Program, Department of Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, USA.
  • Bagel J; Department of Clinical Sciences and Advanced Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, USA.
  • Vite CH; Department of Clinical Sciences and Advanced Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, USA.
  • Bradbury AM; Department of Clinical Sciences and Advanced Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, USA.
  • Wilson JM; Gene Therapy Program, Department of Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, USA.
Hum Gene Ther ; 33(9-10): 499-517, 2022 05.
Article en En | MEDLINE | ID: mdl-35333110

Texto completo: 1 Colección: 01-internacional Banco de datos: MEDLINE Asunto principal: Leucodistrofia de Células Globoides Límite: Animals / Child, preschool / Humans Idioma: En Revista: Hum Gene Ther Asunto de la revista: GENETICA MEDICA / TERAPEUTICA Año: 2022 Tipo del documento: Article País de afiliación: Estados Unidos

Texto completo: 1 Colección: 01-internacional Banco de datos: MEDLINE Asunto principal: Leucodistrofia de Células Globoides Límite: Animals / Child, preschool / Humans Idioma: En Revista: Hum Gene Ther Asunto de la revista: GENETICA MEDICA / TERAPEUTICA Año: 2022 Tipo del documento: Article País de afiliación: Estados Unidos