Successful treatment of severe MSUD in Bckdhb-/- mice with neonatal AAV gene therapy.
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| Title: | Successful treatment of severe MSUD in Bckdhb-/- mice with neonatal AAV gene therapy. |
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| Authors: | 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. |
| Source: | Journal of inherited metabolic disease [J Inherit Metab Dis] 2024 Jan; Vol. 47 (1), pp. 41-49. Date of Electronic Publication: 2023 Mar 15. |
| Publication Type: | Journal Article; Research Support, Non-U.S. Gov't |
| Journal Info: | Publisher: Wiley Country of Publication: United States NLM ID: 7910918 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1573-2665 (Electronic) Linking ISSN: 01418955 NLM ISO Abbreviation: J Inherit Metab Dis Subsets: MEDLINE |
| Database: | MEDLINE Ultimate |
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| ISSN: | 1573-2665 |
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| DOI: | 10.1002/jimd.12604 |
