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

Glycogen storage disease type III (GSDIII) is a rare inborn error of metabolism affecting liver, skeletal muscle, and heart due to mutations of the AGL gene encoding for the glycogen debranching enzyme (GDE). No curative treatment exists for GSDIII. The 4.6 kb GDE cDNA represents the major technical...

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Autores principales:	Antoine Gardin, Jérémy Rouillon, Valle Montalvo-Romeral, Lucille Rossiaud, Patrice Vidal, Romain Launay, Mallaury Vie, Youssef Krimi Benchekroun, Jérémie Cosette, Bérangère Bertin, Tiziana La Bella, Guillaume Dubreuil, Justine Nozi, Louisa Jauze, Romain Fragnoud, Nathalie Daniele, Laetitia Van Wittenberghe, Jérémy Esque, Isabelle André, Xavier Nissan, Lucile Hoch, Giuseppe Ronzitti
Formato:	Artigo
Lenguaje:	Inglês
Publicado:	American Society for Clinical Investigation 2024-01-01
Colección:	The Journal of Clinical Investigation
Materias:	Metabolism Therapeutics
Acceso en línea:	https://doi.org/10.1172/JCI172018
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