Galactosialidosis: preclinical enzyme replacement therapy in a mouse model of the disease, a proof of concept.
Cadaoas J, Hu H, Boyle G, Gomero E, Mosca R, Jayashankar K, Machado M, Cullen S, Guzman B, van de Vlekkert D, Annunziata I, Vellard M, Kakkis E, Koppaka V, d'Azzo A.
Cadaoas J, et al. Among authors: jayashankar k.
Mol Ther Methods Clin Dev. 2020 Nov 20;20:191-203. doi: 10.1016/j.omtm.2020.11.012. eCollection 2021 Mar 12.
Mol Ther Methods Clin Dev. 2020.
PMID: 33426146
Free PMC article.