A functional mini-GDE transgene corrects impairment in models of glycogen storage disease type III.
Gardin A, Rouillon J, Montalvo-Romeral V, Rossiaud L, Vidal P, Launay R, Vie M, Krimi Benchekroun Y, Cosette J, Bertin B, La Bella T, Dubreuil G, Nozi J, Jauze L, Fragnoud R, Daniele N, Van Wittenberghe L, Esque J, André I, Nissan X, Hoch L, Ronzitti G.
Gardin A, et al.
J Clin Invest. 2024 Jan 16;134(2):e172018. doi: 10.1172/JCI172018.
J Clin Invest. 2024.
PMID: 38015640
Free PMC article.
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). ...
Glycogen storage disease type III (GSDIII) is a rare inborn error of metabolism affecting liver, skeletal …