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Successful treatment of severe MSUD in Bckdhb-/- mice with neonatal AAV gene therapy.
Pontoizeau, Clément; Gaborit, Clovis; Tual, Nolan; Simon-Sola, Marcelo; Rotaru, Irina; Benoist, Marion; Colella, Pasqualina; Lamazière, Antonin; Brassier, Anaïs; Arnoux, Jean-Baptiste; Rötig, Agnès; Ottolenghi, Chris; de Lonlay, Pascale; Mingozzi, Federico; Cavazzana, Marina; Schiff, Manuel.
Afiliación
  • Pontoizeau C; Necker Hospital, APHP, Biochemistry, Metabolomics Unit, University Paris Cité, Paris, France.
  • Gaborit C; Necker Hospital, APHP, Reference Center for Inborn Error of Metabolism, Pediatrics Department, University Paris Cité, Paris, France.
  • Tual N; Inserm UMR_S1163, Institut Imagine, Paris, France.
  • Simon-Sola M; Inserm UMR_S1163, Institut Imagine, Paris, France.
  • Rotaru I; Inserm UMR_S1163, Institut Imagine, Paris, France.
  • Benoist M; Inserm UMR_S1163, Institut Imagine, Paris, France.
  • Colella P; Inserm UMR_S1163, Institut Imagine, Paris, France.
  • Lamazière A; Inserm UMR_S1163, Institut Imagine, Paris, France.
  • Brassier A; Généthon INTEGRARE UMR-S951, University of Evry, Evry, France.
  • Arnoux JB; CRSA, St Antoine Hospital, Paris, France.
  • Rötig A; Necker Hospital, APHP, Reference Center for Inborn Error of Metabolism, Pediatrics Department, University Paris Cité, Paris, France.
  • Ottolenghi C; Necker Hospital, APHP, Reference Center for Inborn Error of Metabolism, Pediatrics Department, University Paris Cité, Paris, France.
  • de Lonlay P; Inserm UMR_S1163, Institut Imagine, Paris, France.
  • Mingozzi F; Necker Hospital, APHP, Biochemistry, Metabolomics Unit, University Paris Cité, Paris, France.
  • Cavazzana M; Necker Hospital, APHP, Reference Center for Inborn Error of Metabolism, Pediatrics Department, University Paris Cité, Paris, France.
  • Schiff M; Inserm UMR_S1163, Institut Imagine, Paris, France.
J Inherit Metab Dis ; 47(1): 41-49, 2024 Jan.
Article en En | MEDLINE | ID: mdl-36880392
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Texto completo: 1 Colección: 01-internacional Asunto principal: Enfermedad de la Orina de Jarabe de Arce Límite: Animals / Humans Idioma: En Revista: J Inherit Metab Dis Año: 2024 Tipo del documento: Article País de afiliación: Francia
Texto completo
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Texto completo: 1 Colección: 01-internacional Asunto principal: Enfermedad de la Orina de Jarabe de Arce Límite: Animals / Humans Idioma: En Revista: J Inherit Metab Dis Año: 2024 Tipo del documento: Article País de afiliación: Francia