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. Among authors: la bella t.
J Clin Invest. 2024 Jan 16;134(2):e172018. doi: 10.1172/JCI172018.
J Clin Invest. 2024.
PMID: 38015640
Free PMC article.