A functional mini-GDE transgene corrects impairment in models of glycogen storage disease type III.

Autor: Gardin A; Genethon, Evry, France.; Université Paris-Saclay, Univ Evry, Inserm, Genethon, Integrare research unit UMR_S951, Evry, France., Rouillon J; Genethon, Evry, France.; Université Paris-Saclay, Univ Evry, Inserm, Genethon, Integrare research unit UMR_S951, Evry, France., Montalvo-Romeral V; Genethon, Evry, France.; Université Paris-Saclay, Univ Evry, Inserm, Genethon, Integrare research unit UMR_S951, Evry, France., Rossiaud L; Genethon, Evry, France.; Université Paris-Saclay, Univ Evry, Inserm, Genethon, Integrare research unit UMR_S951, Evry, France.; CECS, I-STEM, Institute for Stem Cell Therapy and Exploration of Monogenic Diseases, Corbeil-Essonnes, France., Vidal P; Genethon, Evry, France.; Université Paris-Saclay, Univ Evry, Inserm, Genethon, Integrare research unit UMR_S951, Evry, France., Launay R; Toulouse Biotechnology Institute, TBI, Université de Toulouse, CNRS, INRAE, INSA, Toulouse, France., Vie M; Genethon, Evry, France.; Université Paris-Saclay, Univ Evry, Inserm, Genethon, Integrare research unit UMR_S951, Evry, France., Krimi Benchekroun Y; Genethon, Evry, France.; Université Paris-Saclay, Univ Evry, Inserm, Genethon, Integrare research unit UMR_S951, Evry, France., Cosette J; Genethon, Evry, France., Bertin B; Genethon, Evry, France.; Université Paris-Saclay, Univ Evry, Inserm, Genethon, Integrare research unit UMR_S951, Evry, France., La Bella T; Genethon, Evry, France.; Université Paris-Saclay, Univ Evry, Inserm, Genethon, Integrare research unit UMR_S951, Evry, France., Dubreuil G; Genethon, Evry, France., Nozi J; Genethon, Evry, France.; Université Paris-Saclay, Univ Evry, Inserm, Genethon, Integrare research unit UMR_S951, Evry, France., Jauze L; Genethon, Evry, France.; Université Paris-Saclay, Univ Evry, Inserm, Genethon, Integrare research unit UMR_S951, Evry, France., Fragnoud R; Genethon, Evry, France., Daniele N; Genethon, Evry, France., Van Wittenberghe L; Genethon, Evry, France., Esque J; Toulouse Biotechnology Institute, TBI, Université de Toulouse, CNRS, INRAE, INSA, Toulouse, France., André I; Toulouse Biotechnology Institute, TBI, Université de Toulouse, CNRS, INRAE, INSA, Toulouse, France., Nissan X; CECS, I-STEM, Institute for Stem Cell Therapy and Exploration of Monogenic Diseases, Corbeil-Essonnes, France., Hoch L; CECS, I-STEM, Institute for Stem Cell Therapy and Exploration of Monogenic Diseases, Corbeil-Essonnes, France., Ronzitti G; Genethon, Evry, France.; Université Paris-Saclay, Univ Evry, Inserm, Genethon, Integrare research unit UMR_S951, Evry, France.

Publikováno v: The Journal of clinical investigation [J Clin Invest] 2024 Jan 16; Vol. 134 (2). Date of Electronic Publication: 2024 Jan 16.