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A functional mini-GDE transgene corrects impairment in models of glycogen storage disease type III.
Gardin, Antoine; Rouillon, Jérémy; Montalvo-Romeral, Valle; Rossiaud, Lucille; Vidal, Patrice; Launay, Romain; Vie, Mallaury; Krimi Benchekroun, Youssef; Cosette, Jérémie; Bertin, Bérangère; La Bella, Tiziana; Dubreuil, Guillaume; Nozi, Justine; Jauze, Louisa; Fragnoud, Romain; Daniele, Nathalie; Van Wittenberghe, Laetitia; Esque, Jérémy; André, Isabelle; Nissan, Xavier; Hoch, Lucile; Ronzitti, Giuseppe.
Afiliação
  • Gardin A; Genethon, Evry, France.
  • Rouillon J; Université Paris-Saclay, Univ Evry, Inserm, Genethon, Integrare research unit UMR_S951, Evry, France.
  • Montalvo-Romeral V; Genethon, Evry, France.
  • Rossiaud L; Université Paris-Saclay, Univ Evry, Inserm, Genethon, Integrare research unit UMR_S951, Evry, France.
  • Vidal P; Genethon, Evry, France.
  • Launay R; Université Paris-Saclay, Univ Evry, Inserm, Genethon, Integrare research unit UMR_S951, Evry, France.
  • Vie M; Genethon, Evry, France.
  • Krimi Benchekroun Y; Université Paris-Saclay, Univ Evry, Inserm, Genethon, Integrare research unit UMR_S951, Evry, France.
  • Cosette J; CECS, I-STEM, Institute for Stem Cell Therapy and Exploration of Monogenic Diseases, Corbeil-Essonnes, France.
  • Bertin B; Genethon, Evry, France.
  • La Bella T; Université Paris-Saclay, Univ Evry, Inserm, Genethon, Integrare research unit UMR_S951, Evry, France.
  • Dubreuil G; Toulouse Biotechnology Institute, TBI, Université de Toulouse, CNRS, INRAE, INSA, Toulouse, France.
  • Nozi J; Genethon, Evry, France.
  • Jauze L; Université Paris-Saclay, Univ Evry, Inserm, Genethon, Integrare research unit UMR_S951, Evry, France.
  • Fragnoud R; Genethon, Evry, France.
  • Daniele N; Université Paris-Saclay, Univ Evry, Inserm, Genethon, Integrare research unit UMR_S951, Evry, France.
  • Van Wittenberghe L; Genethon, Evry, France.
  • Esque J; Genethon, Evry, France.
  • André I; Université Paris-Saclay, Univ Evry, Inserm, Genethon, Integrare research unit UMR_S951, Evry, France.
  • Nissan X; Genethon, Evry, France.
  • Hoch L; Université Paris-Saclay, Univ Evry, Inserm, Genethon, Integrare research unit UMR_S951, Evry, France.
  • Ronzitti G; Genethon, Evry, France.
J Clin Invest ; 134(2)2024 Jan 16.
Article em En | MEDLINE | ID: mdl-38015640

Texto completo: 1 Base de dados: MEDLINE Assunto principal: Sistema da Enzima Desramificadora do Glicogênio / Doença de Depósito de Glicogênio Tipo III Limite: Animals / Humans Idioma: En Ano de publicação: 2024 Tipo de documento: Article

Texto completo: 1 Base de dados: MEDLINE Assunto principal: Sistema da Enzima Desramificadora do Glicogênio / Doença de Depósito de Glicogênio Tipo III Limite: Animals / Humans Idioma: En Ano de publicação: 2024 Tipo de documento: Article