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Successful treatment of severe MSUD in Bckdhb ^-/- mice with neonatal AAV gene therapy.

Autor: Pontoizeau C; Necker Hospital, APHP, Biochemistry, Metabolomics Unit, University Paris Cité, Paris, France.; Necker Hospital, APHP, Reference Center for Inborn Error of Metabolism, Pediatrics Department, University Paris Cité, Paris, France.; Inserm UMR_S1163, Institut Imagine, Paris, France., Gaborit C; Inserm UMR_S1163, Institut Imagine, 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; Généthon INTEGRARE UMR-S951, University of Evry, Evry, France., Lamazière A; CRSA, St Antoine Hospital, Paris, France., Brassier A; Necker Hospital, APHP, Reference Center for Inborn Error of Metabolism, Pediatrics Department, University Paris Cité, Paris, France., Arnoux JB; Necker Hospital, APHP, Reference Center for Inborn Error of Metabolism, Pediatrics Department, University Paris Cité, Paris, France., Rötig A; Inserm UMR_S1163, Institut Imagine, Paris, France., Ottolenghi C; Necker Hospital, APHP, Biochemistry, Metabolomics Unit, University Paris Cité, Paris, France.; Necker Hospital, APHP, Reference Center for Inborn Error of Metabolism, Pediatrics Department, University Paris Cité, Paris, France.; Inserm UMR_S1163, Institut Imagine, Paris, France., de Lonlay P; Necker Hospital, APHP, Reference Center for Inborn Error of Metabolism, Pediatrics Department, University Paris Cité, Paris, France.; Inserm U1151, Institut Necker Enfants Malades, Paris, France., Mingozzi F; Généthon INTEGRARE UMR-S951, University of Evry, Evry, France., Cavazzana M; Inserm UMR_S1163, Institut Imagine, Paris, France.; Necker Hospital, APHP, Biotherapies Department and Clinical Investigation Center, Inserm, University Paris Cité, Paris, France., Schiff M; Necker Hospital, APHP, Reference Center for Inborn Error of Metabolism, Pediatrics Department, University Paris Cité, Paris, France.; Inserm UMR_S1163, Institut Imagine, Paris, France.

Publikováno v: Journal of inherited metabolic disease [J Inherit Metab Dis] 2024 Jan; Vol. 47 (1), pp. 41-49. Date of Electronic Publication: 2023 Mar 15.